Trial Outcomes & Findings for A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (NCT NCT04221451)
NCT ID: NCT04221451
Last Updated: 2026-01-28
Results Overview
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
75 participants
Primary outcome timeframe
Baseline (Day 1) and Week 104
Results posted on
2026-01-28
Participant Flow
This study consisted of 2 periods: primary analysis period (PAP) \& open-label extension (OLE) period. In PAP, primary population (PP) was randomized in a 2:1 ratio to receive either venglustat or placebo in double-blind manner. Secondary population (SP) received OL venglustat. Post completion of PAP, eligible participants entered OLE in which all participants received OL venglustat. The study was early terminated based on absence of positive trends on clinical endpoints; no safety concerns.
In PAP, 59 participants in the PP (adult participants with diagnosis of late-onset disialotetrahexosylganglioside \[GM2\] gangliosidosis) and 16 participants in the SP (juvenile/adolescent participants with diagnosis of late-onset GM2 gangliosidosis, monosialotetrahexosylganglioside \[GM1\] gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) were treated. Randomization for PP was stratified on participant's ability to walk at baseline visit (yes/no).
Participant milestones
| Measure |
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Venglustat
Participants in PP received venglustat 15 milligrams (mg) oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kilograms \[kg\], 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
OLE: PP: Delayed Venglustat
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: PP: Early Venglustat
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: SP: Venglustat
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|---|---|---|
|
PAP: Baseline (Day 1) up to 104 Weeks
STARTED
|
19
|
40
|
16
|
0
|
0
|
0
|
|
PAP: Baseline (Day 1) up to 104 Weeks
COMPLETED
|
17
|
35
|
14
|
0
|
0
|
0
|
|
PAP: Baseline (Day 1) up to 104 Weeks
NOT COMPLETED
|
2
|
5
|
2
|
0
|
0
|
0
|
|
OLE (104 Weeks): From 104 to 208 Weeks
STARTED
|
0
|
0
|
0
|
16
|
34
|
14
|
|
OLE (104 Weeks): From 104 to 208 Weeks
COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
|
OLE (104 Weeks): From 104 to 208 Weeks
NOT COMPLETED
|
0
|
0
|
0
|
16
|
34
|
14
|
Reasons for withdrawal
| Measure |
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Venglustat
Participants in PP received venglustat 15 milligrams (mg) oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kilograms \[kg\], 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
OLE: PP: Delayed Venglustat
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: PP: Early Venglustat
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: SP: Venglustat
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|---|---|---|
|
PAP: Baseline (Day 1) up to 104 Weeks
Withdrawal by Subject
|
2
|
5
|
1
|
0
|
0
|
0
|
|
PAP: Baseline (Day 1) up to 104 Weeks
Other
|
0
|
0
|
1
|
0
|
0
|
0
|
|
OLE (104 Weeks): From 104 to 208 Weeks
Withdrawal by Subject
|
0
|
0
|
0
|
2
|
1
|
2
|
|
OLE (104 Weeks): From 104 to 208 Weeks
Study terminated by sponsor
|
0
|
0
|
0
|
13
|
33
|
12
|
|
OLE (104 Weeks): From 104 to 208 Weeks
Adverse Event
|
0
|
0
|
0
|
1
|
0
|
0
|
Baseline Characteristics
Only SP participants are included for this analysis.
Baseline characteristics by cohort
| Measure |
PAP: PP: Placebo
n=19 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Venglustat
n=40 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=16 Participants
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
Total
n=75 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Clinical diagnosis of SP
Juvenile/adult galactosialidosis
|
—
|
—
|
1 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
1 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
|
Age, Continuous
|
37.8 years
STANDARD_DEVIATION 15.4 • n=19 Participants
|
37.0 years
STANDARD_DEVIATION 13.3 • n=40 Participants
|
12.4 years
STANDARD_DEVIATION 7.3 • n=16 Participants
|
32.0 years
STANDARD_DEVIATION 16.3 • n=75 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=19 Participants
|
19 Participants
n=40 Participants
|
11 Participants
n=16 Participants
|
40 Participants
n=75 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=19 Participants
|
21 Participants
n=40 Participants
|
5 Participants
n=16 Participants
|
35 Participants
n=75 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=19 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=16 Participants
|
0 Participants
n=75 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=19 Participants
|
3 Participants
n=40 Participants
|
2 Participants
n=16 Participants
|
5 Participants
n=75 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=19 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=16 Participants
|
0 Participants
n=75 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=19 Participants
|
0 Participants
n=40 Participants
|
1 Participants
n=16 Participants
|
1 Participants
n=75 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=19 Participants
|
35 Participants
n=40 Participants
|
12 Participants
n=16 Participants
|
64 Participants
n=75 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=19 Participants
|
0 Participants
n=40 Participants
|
0 Participants
n=16 Participants
|
0 Participants
n=75 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=19 Participants
|
2 Participants
n=40 Participants
|
1 Participants
n=16 Participants
|
5 Participants
n=75 Participants
|
|
PP: Cerebrospinal fluid (CSF) GM2 biomarker
|
67.11 nanogram/milliliter (ng/mL)
STANDARD_DEVIATION 34.15 • n=18 Participants • PAP: Primary pharmacodynamic (PD) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those PP participants with data collected at baseline are reported.
|
63.00 nanogram/milliliter (ng/mL)
STANDARD_DEVIATION 36.84 • n=39 Participants • PAP: Primary pharmacodynamic (PD) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those PP participants with data collected at baseline are reported.
|
—
|
64.30 nanogram/milliliter (ng/mL)
STANDARD_DEVIATION 35.76 • n=57 Participants • PAP: Primary pharmacodynamic (PD) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those PP participants with data collected at baseline are reported.
|
|
PP: 9-hole peg Test (9-HPT)
|
45.89 seconds
STANDARD_DEVIATION 34.65 • n=19 Participants • Only PP participants are included for this analysis.
|
41.93 seconds
STANDARD_DEVIATION 26.56 • n=40 Participants • Only PP participants are included for this analysis.
|
—
|
43.20 seconds
STANDARD_DEVIATION 29.16 • n=59 Participants • Only PP participants are included for this analysis.
|
|
Clinical diagnosis of SP
Juvenile/adolescent GM2 gangliosidosis
|
—
|
—
|
7 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
7 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
|
Clinical diagnosis of SP
GM1 gangliosidosis
|
—
|
—
|
7 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
7 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
|
Clinical diagnosis of SP
Saposin C deficiency
|
—
|
—
|
0 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
0 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
|
Clinical diagnosis of SP
Sialidosis type 1
|
—
|
—
|
1 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
1 Participants
n=16 Participants • Only SP participants are included for this analysis.
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=31 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=16 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP: Percent Change From Baseline in CSF GM2 Biomarker to Week 104
|
-47.57 percent change
Standard Error 3.04
|
-11.33 percent change
Standard Error 4.23
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP: Primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years. Only those participants with data collected at specified timepoints are reported.
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs and block containing 9 empty holes.On start command when stopwatch is started,participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes and once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container. Both dominant and non-dominant hands are tested twice (2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand).Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value on 9-HPT is indicative of higher disability.Mean annualized rate of change in 9-HPT was obtained from exponential transformation of mean slope of log-transformed 9-HPT.Baseline: last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=39 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=18 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP: Annualized Rate of Change From Baseline in the 9-HPT to Week 104
|
2.49 percent per year
Standard Error 1.35
|
0.95 percent per year
Standard Error 1.92
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP: Analysis was performed on juvenile/adolescent late-onset GM2 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD.
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM2 biomarkers in juvenile/adolescent late-onset GM2 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=7 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
GL-1: CSF
|
—
|
-82.35 percent change
Standard Deviation 9.94
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
GL-1: Plasma
|
—
|
-79.28 percent change
Standard Deviation 2.56
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
GM2: CSF
|
—
|
-43.41 percent change
Standard Deviation 23.14
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
GM2: Plasma
|
—
|
-55.91 percent change
Standard Deviation 11.76
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP: Analysis was performed on GM1 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM1 biomarkers in GM1 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=6 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
GL-1: Plasma
|
—
|
-80.42 percent change
Standard Deviation 8.17
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
GM1: CSF
|
—
|
-32.35 percent change
Standard Deviation 17.46
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
GL-1: CSF
|
—
|
17.42 percent change
Standard Deviation 188.41
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
GM1: Plasma
|
—
|
-52.32 percent change
Standard Deviation 16.76
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM2 and GM3 biomarkers in sialidosis type 1 participant of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on sialidosis type 1 participant from secondary PD population (all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD).
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=1 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GL-1: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GL-1: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GM2: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GM2: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GM3: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
GM3: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM1 and GM3 biomarkers in juvenile/adult galactosialidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on juvenile/adult galactosialidosis participant from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=1 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GL-1: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GL-1: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GM1: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GM1: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GM3: CSF
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
|
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
GM3: Plasma
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1) to Week 104Population: PAP: Analysis was planned to be performed on saposin C deficiency participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. As presented in baseline characteristics, there was no participant enrolled with Saposin C deficiency.
Plasma and CSF samples were planned to be collected at specified timepoints to assess the presence of GL-1 biomarkers in saposin C deficiency participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=31 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=16 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP: Absolute Change From Baseline in CSF GM2 Biomarker to Week 104
|
-32.83 ng/mL
Standard Error 1.70
|
-17.64 ng/mL
Standard Error 2.36
|
—
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: PAP PP:primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years.PAP SP: secondary efficacy population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.Only those participants with ability to walk at baseline and data collected at specified timepoints are reported.
The 25FWT is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly and safely as possible for 2 trials (can use assistive devices). The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The amount of time (in seconds) to walk 25 feet is recorded (ranging up to 180 seconds). The 25FWT score is defined as the average of 2 trials. A higher value on the 25FWT is indicative of higher disability. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=30 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=16 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=11 Participants
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104
PP
|
8.9 seconds
Standard Deviation 31.0
|
1.4 seconds
Standard Deviation 4.2
|
—
|
|
PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104
SP: juvenile/adolescent late-onset GM2 gangliodosis
|
—
|
—
|
29.6 seconds
Standard Deviation 66.5
|
|
PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104
SP: GM1 gangliosidosis
|
—
|
—
|
0.4 seconds
Standard Deviation 0.8
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
The FARS-neuro includes 23 items and is composed of 4 sections that assesses different neurological faculties: bulbar activity (4 items), upper limb coordination (5 items assessed right and left side), lower limb coordination (2 items assessed right and left side), peripheral nervous system (5 items assessed right and left side) and upright stability (7 items). Total score is calculated as the sum of scores on items of this section and ranges from 0 to 117; mean is presented. Higher value indicates higher disability. Baseline=last available value before or equal to first dose of study drug date in PAP. PP: primary efficacy population: all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years. SP: secondary efficacy population: all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=35 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=17 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=13 Participants
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
PP
|
-1.0 score on a scale
Standard Deviation 8.5
|
0.4 score on a scale
Standard Deviation 6.0
|
—
|
|
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
SP: juvenile/adolescent late-onset GM2 gangliodosis
|
—
|
—
|
2.8 score on a scale
Standard Deviation 10.6
|
|
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
SP: GM1 gangliosidosis
|
—
|
—
|
-2.5 score on a scale
Standard Deviation 17.0
|
|
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
SP: sialidosis type 1
|
—
|
—
|
NA score on a scale
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality.
|
SECONDARY outcome
Timeframe: From first dose of study drug (Day 1) up to end of PAP, 104 weeksPopulation: PAP PP: primary safety population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug. PAP SP: secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug.
An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAE were AEs that developed, worsened or became serious during the TE period.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=40 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=19 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=16 Participants
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
TEAEs
|
40 Participants
|
19 Participants
|
14 Participants
|
|
PAP: PP and SP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
TESAEs
|
8 Participants
|
4 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12Population: PAP: Primary pharmacokinetic (PK) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years who received at least 1 dose of study drug and had at least 1 PK assessment. Only those participants with data collected at specified timepoints are reported.
Plasma samples were collected at specified timepoints to obtain venglustat concentrations for PP in PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=30 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP: Plasma Venglustat Concentration
0 hour
|
—
|
112 ng/mL
Standard Deviation 43.7
|
—
|
|
PAP: PP: Plasma Venglustat Concentration
0.5 hours
|
—
|
125 ng/mL
Standard Deviation 54.1
|
—
|
|
PAP: PP: Plasma Venglustat Concentration
3 hours
|
—
|
188 ng/mL
Standard Deviation 60.8
|
—
|
|
PAP: PP: Plasma Venglustat Concentration
8 hours
|
—
|
149 ng/mL
Standard Deviation 47.3
|
—
|
|
PAP: PP: Plasma Venglustat Concentration
12 hours
|
—
|
132 ng/mL
Standard Deviation 44.5
|
—
|
|
PAP: PP: Plasma Venglustat Concentration
24 hours
|
—
|
105 ng/mL
Standard Deviation 38.5
|
—
|
SECONDARY outcome
Timeframe: Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12Population: PAP: Secondary PK population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug and had at least 1 PK assessment. 'Overall Number of Participants Analyzed' indicates the total of maximum number of participants ('number analyzed') for each dose.
Plasma samples were collected at specified timepoints to obtain venglustat concentration for SP in PAP. Data is presented by dose level for all diseases combined for SP in PAP.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=15 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 8 hours
|
—
|
96.4 ng/mL
Standard Deviation 10.2
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 12 hours
|
—
|
88.9 ng/mL
Standard Deviation 6.05
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 24 hours
|
—
|
64.5 ng/mL
Standard Deviation 13.5
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 3 hours
|
—
|
112 ng/mL
Standard Deviation 40.8
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 8 hours
|
—
|
98.2 ng/mL
Standard Deviation 22.0
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 12 hours
|
—
|
79.3 ng/mL
Standard Deviation 35.8
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 24 hours
|
—
|
49.5 ng/mL
Standard Deviation 17.4
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 0 hour
|
—
|
67.7 ng/mL
Standard Deviation 7.05
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 1.5 hours
|
—
|
86.7 ng/mL
Standard Deviation 35.0
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
15 mg: 3 hours
|
—
|
124 ng/mL
Standard Deviation 16.0
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
4 mg: 0 hour
|
—
|
56.4 ng/mL
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
4 mg: 3 hours
|
—
|
144 ng/mL
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
4 mg: 8 hours
|
—
|
101 ng/mL
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
4 mg: 12 hours
|
—
|
92.3 ng/mL
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
4 mg: 24 hours
|
—
|
58.1 ng/mL
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 0 hour
|
—
|
61.5 ng/mL
Standard Deviation 34.1
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 0.5 hours
|
—
|
85.9 ng/mL
Standard Deviation 56.8
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 3 hours
|
—
|
127 ng/mL
Standard Deviation 72.4
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 8 hours
|
—
|
97.9 ng/mL
Standard Deviation 55.6
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 12 hours
|
—
|
87.9 ng/mL
Standard Deviation 55.9
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
6 mg: 24 hours
|
—
|
50.2 ng/mL
Standard Deviation 25.5
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 0 hour
|
—
|
58.4 ng/mL
Standard Deviation 40.8
|
—
|
|
PAP: SP: Plasma Venglustat Concentration
12 mg: 0.5 hours
|
—
|
67.4 ng/mL
Standard Deviation 36.4
|
—
|
SECONDARY outcome
Timeframe: Week 104Population: PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment.
CSF samples were collected via lumbar puncture at Week 104 to obtain venglustat concentrations for PP and SP in PAP. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=7 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=18 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: CSF Venglustat Concentration
PP
|
—
|
5.64 ng/mL
Standard Deviation 2.05
|
—
|
|
PAP: PP and SP: CSF Venglustat Concentration
SP: 6 mg
|
3.37 ng/mL
Standard Deviation 2.19
|
—
|
—
|
|
PAP: PP and SP: CSF Venglustat Concentration
SP: 12 mg
|
3.22 ng/mL
Standard Deviation 1.01
|
—
|
—
|
|
PAP: PP and SP: CSF Venglustat Concentration
SP: 15 mg
|
5.41 ng/mL
Standard Deviation 0.57
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12Population: PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment.
Plasma samples were collected at specified timepoints to obtain Cmax of venglustat. The mean of Cmax, irrespective of the timepoint where the patient-individual Cmax value was observed is presented as opposed to endpoint 13 wherein mean of plasma venglustat concentration at each specified timepoint is presented. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=15 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=30 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
PP
|
—
|
188 ng/mL
Standard Deviation 60.8
|
—
|
|
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
SP: 4 mg
|
144 ng/mL
|
—
|
—
|
|
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
SP: 6 mg
|
127 ng/mL
Standard Deviation 72.4
|
—
|
—
|
|
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
SP: 12 mg
|
121 ng/mL
Standard Deviation 39.5
|
—
|
—
|
|
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
SP: 15 mg
|
124 ng/mL
Standard Deviation 16.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12Population: PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment.
Plasma samples were collected at specified timepoints to obtain tmax of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=15 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=30 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
PP
|
—
|
3.00 hour
Interval 2.77 to 7.05
|
—
|
|
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
SP: 4 mg
|
3.50 hour
|
—
|
—
|
|
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
SP: 6 mg
|
3.00 hour
Interval 2.25 to 3.02
|
—
|
—
|
|
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
SP: 12 mg
|
5.50 hour
Interval 3.0 to 12.0
|
—
|
—
|
|
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
SP: 15 mg
|
3.00 hour
Interval 3.0 to 3.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12Population: PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged \>=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug \& had at least 1 PK assessment.
Plasma samples were collected at specified timepoints to obtain AUC0-24 of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Outcome measures
| Measure |
PAP: PP: Venglustat
n=12 Participants
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=22 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
PP
|
—
|
3230 hour*ng/mL
Standard Deviation 1110
|
—
|
|
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
SP: 4 mg
|
2170 hour*ng/mL
|
—
|
—
|
|
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
SP: 6 mg
|
1600 hour*ng/mL
Standard Deviation 765
|
—
|
—
|
|
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
SP: 12 mg
|
1840 hour*ng/mL
Standard Deviation 415
|
—
|
—
|
|
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
SP: 15 mg
|
2150 hour*ng/mL
Standard Deviation 275
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Week 104Population: Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs \& block containing 9 empty holes.On start command (stopwatch started),participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes \& once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container,2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand.Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value indicates higher disability.Baseline: last available value before or equal to first dose of study drug date in PAP.Secondary efficacy population:all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis,GM1 gangliosidosis,saposin C deficiency,sialidosis type 1 or juvenile/adult galactosialidosis.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=11 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104
Juvenile/adolescent late-onset GM2 gangliodosis
|
—
|
-1.2 percent change
Standard Error 24.5
|
—
|
|
PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104
GM1 gangliosidosis
|
—
|
11.7 percent change
Standard Error 34.9
|
—
|
|
PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104
Sialidosis type 1
|
—
|
NA percent change
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality.
|
—
|
SECONDARY outcome
Timeframe: Baseline (Day 1) to Week 104Population: Secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug. Only pediatric participants were evaluated for this endpoint.
For the secondary pediatric population, the acceptability and palatability of venglustat tablets was assessed through the route of venglustat administration collected in the electronic case report form and study drug compliance throughout PAP. The assessment was based on tablet always swallowed as whole or chewed and swallowed at least once. Number of participants with \>=80% compliance for each is presented here.
Outcome measures
| Measure |
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Placebo
n=13 Participants
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|
|
PAP: SP: Number of Participants With >=80% Compliance as Per Method of Intake
Participants always swallowed the tablet as whole
|
—
|
8 Participants
|
—
|
|
PAP: SP: Number of Participants With >=80% Compliance as Per Method of Intake
Participants chewed and swallowed the tablet at least once
|
—
|
4 Participants
|
—
|
Adverse Events
PAP: PP: Placebo
Serious events: 4 serious events
Other events: 19 other events
Deaths: 0 deaths
PAP: PP: Venglustat
Serious events: 8 serious events
Other events: 38 other events
Deaths: 0 deaths
PAP: SP: Venglustat
Serious events: 6 serious events
Other events: 14 other events
Deaths: 1 deaths
OLE: PP: Delayed Venglustat
Serious events: 4 serious events
Other events: 11 other events
Deaths: 1 deaths
OLE: PP: Early Venglustat
Serious events: 2 serious events
Other events: 19 other events
Deaths: 0 deaths
OLE: SP: Venglustat
Serious events: 2 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PAP: PP: Placebo
n=19 participants at risk
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Venglustat
n=40 participants at risk
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=16 participants at risk
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
OLE: PP: Delayed Venglustat
n=16 participants at risk
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: PP: Early Venglustat
n=34 participants at risk
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: SP: Venglustat
n=14 participants at risk
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|---|---|---|
|
Infections and infestations
Burn Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Cellulitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Cystitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Infected Bite
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Respiratory Tract Infection Viral
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Sepsis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Urinary Tract Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Metabolism and nutrition disorders
Hypophagia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Acute Psychosis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Completed Suicide
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Mania
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Persecutory Delusion
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Suicidal Ideation
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Dyskinesia
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Faecaloma
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Oesophageal Achalasia
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Hepatobiliary disorders
Hepatic Mass
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Intervertebral Disc Protrusion
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Urinary Retention
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Ankle Fracture
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Femur Fracture
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Hip Fracture
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Post Lumbar Puncture Syndrome
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Tibia Fracture
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
Other adverse events
| Measure |
PAP: PP: Placebo
n=19 participants at risk
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: PP: Venglustat
n=40 participants at risk
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
|
PAP: SP: Venglustat
n=16 participants at risk
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
OLE: PP: Delayed Venglustat
n=16 participants at risk
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: PP: Early Venglustat
n=34 participants at risk
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
|
OLE: SP: Venglustat
n=14 participants at risk
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if \>=50 kg, 12 mg if 30 kg to \<50 kg, 6 mg if 15 kg to \<30 kg and 4 mg if 10 kg to \<15 kg.
|
|---|---|---|---|---|---|---|
|
Infections and infestations
Bacterial Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Bacterial Vaginosis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Breast Abscess
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Bronchiolitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Bronchitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Covid-19
|
15.8%
3/19 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
32.5%
13/40 • Number of events 14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
43.8%
7/16 • Number of events 8 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.9%
2/34 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Conjunctivitis
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Enterobiasis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 7 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Fungal Foot Infection
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Gastroenteritis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Helicobacter Gastritis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Herpes Zoster
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Hordeolum
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Infected Bite
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Influenza
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Kidney Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Localised Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Nasopharyngitis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
31.2%
5/16 • Number of events 13 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Oral Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Otitis Media
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Perichondritis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Pharyngotonsillitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Pneumonia
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
8.8%
3/34 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 6 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Sinusitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Skin Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Urinary Tract Infection
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
15.0%
6/40 • Number of events 9 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
8.8%
3/34 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
14.3%
2/14 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Urinary Tract Infection Bacterial
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Vulvovaginal Candidiasis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Infections and infestations
Vulvovaginal Mycotic Infection
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Immune system disorders
Drug Hypersensitivity
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Immune system disorders
Seasonal Allergy
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Metabolism and nutrition disorders
Cachexia
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Metabolism and nutrition disorders
Decreased Appetite
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Metabolism and nutrition disorders
Iron Deficiency
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Metabolism and nutrition disorders
Vitamin D Deficiency
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Anxiety
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 9 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Apathy
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Depression
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Disorientation
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Emotional Disorder
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Grief Reaction
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Irritability
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.9%
2/34 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Psychiatric disorders
Mania
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Ataxia
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Balance Disorder
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Disturbance In Attention
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.9%
2/34 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Dizziness
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Headache
|
21.1%
4/19 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
25.0%
10/40 • Number of events 19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
25.0%
4/16 • Number of events 10 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
8.8%
3/34 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 9 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Hypoaesthesia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Hyporeflexia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Hypotonia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Loss Of Consciousness
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Myasthenia Gravis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Myoclonus
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Paraesthesia
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Seizure
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Speech Disorder
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Nervous system disorders
Tremor
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Blepharitis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Cataract
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Cataract Cortical
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Cataract Nuclear
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Cataract Subcapsular
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Dry Eye
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Eyelid Ptosis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Lenticular Opacities
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Meibomianitis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Strabismus
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Vision Blurred
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Visual Field Defect
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Eye disorders
Visual Impairment
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Ear and labyrinth disorders
Ear Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Cardiac disorders
Tachycardia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
20.0%
8/40 • Number of events 8 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Obstructive Sleep Apnoea Syndrome
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis Allergic
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Tract Congestion
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Colitis Ulcerative
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Constipation
|
15.8%
3/19 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 6 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.9%
2/34 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Dental Caries
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Diarrhoea
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
5/40 • Number of events 8 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Duodenogastric Reflux
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Dysphagia
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Gastritis Erosive
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Gastrooesophageal Reflux Disease
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Nausea
|
15.8%
3/19 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
17.5%
7/40 • Number of events 10 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Tongue Ulceration
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Tooth Impacted
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Gastrointestinal disorders
Vomiting
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 5 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
18.8%
3/16 • Number of events 5 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Dermatitis Allergic
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Eczema
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Hand Dermatitis
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Night Sweats
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Pruritus Allergic
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic Dermatitis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Skin Exfoliation
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
15.8%
3/19 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
17.5%
7/40 • Number of events 8 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
18.8%
3/16 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
15.8%
3/19 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Joint Swelling
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Muscular Weakness
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Chest Pain
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Neck Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Musculoskeletal and connective tissue disorders
Pain In Extremity
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
5/40 • Number of events 5 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Crystalluria
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Nephrolithiasis
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Pollakiuria
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Renal and urinary disorders
Urinary Retention
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.0%
2/40 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Reproductive system and breast disorders
Amenorrhoea
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Reproductive system and breast disorders
Cervical Polyp
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Chills
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Face Oedema
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Fatigue
|
15.8%
3/19 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Gait Disturbance
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Injection Site Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Medical Device Site Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Pyrexia
|
15.8%
3/19 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.5%
3/40 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
General disorders
Vaccination Site Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Alanine Aminotransferase Increased
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
5.9%
2/34 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Blood Urine Present
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Depression Rating Scale Score Increased
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Electrocardiogram Qt Prolonged
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Gamma-Glutamyltransferase Increased
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Hepatic Enzyme Increased
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Lymphocyte Count Increased
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Muscle Strength Abnormal
|
5.3%
1/19 • Number of events 8 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Platelet Count Decreased
|
5.3%
1/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
Weight Decreased
|
15.8%
3/19 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Investigations
White Blood Cell Count Increased
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Accidental Overdose
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 7 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Bone Contusion
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Contusion
|
21.1%
4/19 • Number of events 6 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
22.5%
9/40 • Number of events 10 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Face Injury
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Fall
|
52.6%
10/19 • Number of events 31 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
55.0%
22/40 • Number of events 61 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 3 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
11.8%
4/34 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Fibula Fracture
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Foot Fracture
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Head Injury
|
10.5%
2/19 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Immunisation Reaction
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
7.1%
1/14 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Ligament Sprain
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 6 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Muscle Strain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Post Lumbar Puncture Syndrome
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
10.0%
4/40 • Number of events 4 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
12.5%
2/16 • Number of events 2 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Procedural Pain
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Scratch
|
5.3%
1/19 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/40 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/16 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/34 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
|
Injury, poisoning and procedural complications
Skin Laceration
|
0.00%
0/19 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.5%
1/40 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
6.2%
1/16 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
2.9%
1/34 • Number of events 1 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
0.00%
0/14 • Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
|
Additional Information
Trial Transparency Team
Sanofi aventis recherche & développement
Phone: 800-633-1610
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
- Publication restrictions are in place
Restriction type: OTHER