Trial Outcomes & Findings for A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) (NCT NCT00891202)
NCT ID: NCT00891202
Last Updated: 2017-03-03
Results Overview
Percent change in spleen volume = (\[spleen volume at Week 39 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
40 participants
Primary outcome timeframe
PAP Baseline (Day 1), Week 39
Results posted on
2017-03-03
Participant Flow
A total of 72 participants were screened between 5 November 2009 and 29 July 2011, of which 32 participants were screen failure. Overall 40 participants were enrolled and the study was conducted across 18 centers in 12 countries.
The 40 participants who met inclusion criteria received placebo or Genz-112638 (eliglustat tartrate) during 39 weeks primary analysis period (PAP). After Week 39 of the PAP, all participants who remained in the study received eliglustat tartrate in the long-term treatment period (LTTP) for up to Week 312.
Participant milestones
| Measure |
PAP: Eliglustat
Eliglustat tartrate capsule as a single 50 milligram (mg) dose on Day 1 followed by eliglustat tartrate 50 mg capsule twice daily (BID) from Day 2 to Week 4, and then either eliglustat tartrate 50 mg capsule BID (in participants who had a Genz-99067 \[active moiety of eliglustat tartrate in plasma\] trough plasma concentration greater than or equal to \[\>=\] 5 nanogram per milliliter \[ng/mL\]) or eliglustat tartrate 100 mg capsule BID (in participants who had a Genz-99067 trough plasma concentration less than \[\<\] 5 ng/mL), up to Week 39. The pharmacokinetic (PK) assessment at Week 2 was used for dose adjustment after Week 4.
|
PAP: Placebo
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
LTTP: Eliglustat (Originally on Eliglustat)
Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
LTTP: Eliglustat (Originally on Placebo)
Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
|---|---|---|---|---|
|
PAP (Up To Week 39)
STARTED
|
20
|
20
|
0
|
0
|
|
PAP (Up To Week 39)
COMPLETED
|
19
|
20
|
0
|
0
|
|
PAP (Up To Week 39)
NOT COMPLETED
|
1
|
0
|
0
|
0
|
|
LTTP (Post-Week 39 up to Week 312)
STARTED
|
0
|
0
|
19
|
20
|
|
LTTP (Post-Week 39 up to Week 312)
COMPLETED
|
0
|
0
|
12
|
15
|
|
LTTP (Post-Week 39 up to Week 312)
NOT COMPLETED
|
0
|
0
|
7
|
5
|
Reasons for withdrawal
| Measure |
PAP: Eliglustat
Eliglustat tartrate capsule as a single 50 milligram (mg) dose on Day 1 followed by eliglustat tartrate 50 mg capsule twice daily (BID) from Day 2 to Week 4, and then either eliglustat tartrate 50 mg capsule BID (in participants who had a Genz-99067 \[active moiety of eliglustat tartrate in plasma\] trough plasma concentration greater than or equal to \[\>=\] 5 nanogram per milliliter \[ng/mL\]) or eliglustat tartrate 100 mg capsule BID (in participants who had a Genz-99067 trough plasma concentration less than \[\<\] 5 ng/mL), up to Week 39. The pharmacokinetic (PK) assessment at Week 2 was used for dose adjustment after Week 4.
|
PAP: Placebo
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
LTTP: Eliglustat (Originally on Eliglustat)
Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
LTTP: Eliglustat (Originally on Placebo)
Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline of LTTP for this arm. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
|---|---|---|---|---|
|
PAP (Up To Week 39)
Withdrawal by Subject
|
1
|
0
|
0
|
0
|
|
LTTP (Post-Week 39 up to Week 312)
Withdrawal by Subject
|
0
|
0
|
4
|
0
|
|
LTTP (Post-Week 39 up to Week 312)
Pregnancy
|
0
|
0
|
1
|
0
|
|
LTTP (Post-Week 39 up to Week 312)
Transitioned to commercial eliglustat
|
0
|
0
|
2
|
5
|
Baseline Characteristics
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Baseline characteristics by cohort
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
Total
n=40 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
31.6 years
STANDARD_DEVIATION 11.55 • n=99 Participants
|
32.1 years
STANDARD_DEVIATION 11.26 • n=107 Participants
|
31.8 years
STANDARD_DEVIATION 11.26 • n=206 Participants
|
|
Gender
Female
|
12 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Gender
Male
|
8 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Race: White
|
19 participants
n=99 Participants
|
20 participants
n=107 Participants
|
39 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Race: Asian
|
1 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Not Hispanic or Latino
|
18 participants
n=99 Participants
|
20 participants
n=107 Participants
|
38 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Ethnicity: Hispanic or Latino
|
2 participants
n=99 Participants
|
0 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Body Mass Index (BMI)
|
23.3 kilogram per square meter (kg/m^2)
STANDARD_DEVIATION 2.74 • n=99 Participants
|
23.4 kilogram per square meter (kg/m^2)
STANDARD_DEVIATION 3.54 • n=107 Participants
|
23.4 kilogram per square meter (kg/m^2)
STANDARD_DEVIATION 3.13 • n=206 Participants
|
|
Weight
|
64.8 kilogram (kg)
STANDARD_DEVIATION 11.74 • n=99 Participants
|
68.6 kilogram (kg)
STANDARD_DEVIATION 17.17 • n=107 Participants
|
66.7 kilogram (kg)
STANDARD_DEVIATION 14.65 • n=206 Participants
|
|
Height
|
166.2 centimeter (cm)
STANDARD_DEVIATION 9.91 • n=99 Participants
|
170.0 centimeter (cm)
STANDARD_DEVIATION 12.02 • n=107 Participants
|
168.1 centimeter (cm)
STANDARD_DEVIATION 11.05 • n=206 Participants
|
PRIMARY outcome
Timeframe: PAP Baseline (Day 1), Week 39Population: FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Percent change in spleen volume = (\[spleen volume at Week 39 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN.
Outcome measures
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo
|
-27.77 percent change
Standard Error 2.37
|
2.26 percent change
Standard Error 2.37
|
SECONDARY outcome
Timeframe: PAP Baseline (Day 1)Population: FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Outcome measures
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
PAP: Hemoglobin Level
|
12.05 gram per deciliter (g/dL)
Standard Deviation 1.816
|
12.75 gram per deciliter (g/dL)
Standard Deviation 1.629
|
SECONDARY outcome
Timeframe: PAP Baseline (Day 1), Week 39Population: FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.
Outcome measures
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
PAP: Absolute Change From Baseline in Hemoglobin Level at Week 39
|
0.69 g/dL
Standard Error 0.23
|
-0.54 g/dL
Standard Error 0.23
|
SECONDARY outcome
Timeframe: PAP Baseline (Day 1), Week 39Population: FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Percent change in liver volume = (\[liver volume at Week 39 minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in MN.
Outcome measures
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
PAP: Percent Change From Baseline in Liver Volume (in MN) at Week 39
|
-5.20 percent change
Standard Error 1.64
|
1.44 percent change
Standard Error 1.64
|
SECONDARY outcome
Timeframe: PAP Baseline (Day 1), Week 39Population: FAS for PAP included all participants who signed informed consent and received at least one dose of study drug (placebo or eliglustat).
Percent change in platelet count = (\[platelet count at Week 39 minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100.
Outcome measures
| Measure |
PAP: Eliglustat
n=20 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=20 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
PAP: Percent Change From Baseline in Platelet Counts at Week 39
|
32.00 percent change
Standard Error 5.95
|
-9.06 percent change
Standard Error 5.95
|
SECONDARY outcome
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234Population: Intent-to-treat (ITT) population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed= participants evaluable for this outcome measure and had available data for baseline and Week 234 spleen volume assessment.
Percent change in spleen volume = (\[spleen volume at Week 234 minus spleen volume at baseline\] divided by \[spleen volume at baseline\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.
Outcome measures
| Measure |
PAP: Eliglustat
n=7 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=6 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
LTTP: Percent Change From Baseline in Spleen Volume (in MN) at Week 234
|
-66.9 percent change
Standard Deviation 8.45
|
-64.0 percent change
Standard Deviation 6.43
|
SECONDARY outcome
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234Population: ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 hemoglobin level assessment.
Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.
Outcome measures
| Measure |
PAP: Eliglustat
n=7 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=5 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
LTTP: Absolute Change From Baseline in Hemoglobin Level at Week 234
|
1.1 g/dL
Standard Deviation 0.65
|
1.9 g/dL
Standard Deviation 1.88
|
SECONDARY outcome
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234Population: ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 liver volume assessment.
Percent change in liver volume = (\[liver volume at Week 234 minus liver volume at baseline\] divided by \[liver volume at baseline\]) multiplied by 100, where all volumes are in MN. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.
Outcome measures
| Measure |
PAP: Eliglustat
n=7 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=6 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
LTTP: Percent Change From Baseline in Liver Volume (in MN) at Week 234
|
-24.3 percent change
Standard Deviation 11.21
|
-22.4 percent change
Standard Deviation 10.77
|
SECONDARY outcome
Timeframe: PAP Baseline for Eliglustat (Originally on Eliglustat) arm, LTTP Baseline for Eliglustat (Originally on Placebo) arm, Week 234Population: ITT population for LTTP included all participants who received at least 1 dose of eliglustat in LTTP period. Number of participants analyzed=participants evaluable for this outcome measure and had available data for baseline and Week 234 platelet count assessment.
Percent change in platelet count = (\[platelet count at Week 234 minus platelet count at baseline\] divided by \[platelet count at baseline\]) multiplied by 100. Baseline values for the original placebo participants refer to Day 1 of LTTP and baseline values for the original eliglustat participants refer to the Day 1 of PAP.
Outcome measures
| Measure |
PAP: Eliglustat
n=7 Participants
Eliglustat tartrate 50 mg capsule BID orally from Day 1 to Week 4, followed by eliglustat tartrate 50 mg or 100 mg capsule BID orally up to Week 39.
|
PAP: Placebo
n=5 Participants
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
|
|---|---|---|
|
LTTP: Percent Change From Baseline in Platelet Counts at Week 234
|
77.3 percent change
Standard Deviation 28.17
|
100.1 percent change
Standard Deviation 80.69
|
Adverse Events
Eliglustat
Serious events: 2 serious events
Other events: 17 other events
Deaths: 0 deaths
Placebo
Serious events: 3 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eliglustat
n=20 participants at risk
PAP: Eliglustat tartrate capsule 50 mg orally on Day 1 followed by eliglustat tartrate 50 mg capsule BID from Day 2 to Week 4, then either eliglustat tartrate 50 mg capsule BID(participants with Genz-99067 trough plasma concentration\>=5 ng/mL) or eliglustat tartrate 100 mg capsule BID(participants with Genz-99067 trough plasma concentration\<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP \& received eliglustat tartrate capsule 50 mg BID orally from Day 1(post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 \& Week 47 were based on Genz-99067 trough plasma concentrations(if trough plasma concentration\<5 ng/mL: next higher dose administered;if\>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
Placebo
n=20 participants at risk
PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
|---|---|---|
|
Cardiac disorders
Atrioventricular block
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Cardiac disorders
Atrioventricular block second degree
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Cardiac disorders
Ventricular tachycardia
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Hepatobiliary disorders
Biliary colic
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Appendicitis
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
Other adverse events
| Measure |
Eliglustat
n=20 participants at risk
PAP: Eliglustat tartrate capsule 50 mg orally on Day 1 followed by eliglustat tartrate 50 mg capsule BID from Day 2 to Week 4, then either eliglustat tartrate 50 mg capsule BID(participants with Genz-99067 trough plasma concentration\>=5 ng/mL) or eliglustat tartrate 100 mg capsule BID(participants with Genz-99067 trough plasma concentration\<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP \& received eliglustat tartrate capsule 50 mg BID orally from Day 1(post Week 39) until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 \& Week 47 were based on Genz-99067 trough plasma concentrations(if trough plasma concentration\<5 ng/mL: next higher dose administered;if\>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
Placebo
n=20 participants at risk
PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
|
|---|---|---|
|
Nervous system disorders
Dizziness
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Nervous system disorders
Headache
|
55.0%
11/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
35.0%
7/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Nervous system disorders
Migraine
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Psychiatric disorders
Anxiety
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Renal and urinary disorders
Proteinuria
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
General disorders
Fatigue
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Cardiac disorders
Palpitations
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Eye disorders
Eye irritation
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Abdominal distension
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
5/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Dry mouth
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Dyspepsia
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Gastritis
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Nausea
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Toothache
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
General disorders
Asthenia
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
General disorders
Oedema peripheral
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
General disorders
Pyrexia
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Immune system disorders
Seasonal allergy
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Bronchitis
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Gastroenteritis
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Hordeolum
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Nasopharyngitis
|
25.0%
5/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Otitis media
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Sinusitis
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Tonsillitis
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Upper respiratory tract infection
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Infections and infestations
Urinary tract infection
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Injury, poisoning and procedural complications
Contusion
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Investigations
Blood creatine phosphokinase increased
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Investigations
Bone density decreased
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
55.0%
11/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
20.0%
4/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
5/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
15.0%
3/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
5.0%
1/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
10.0%
2/20 • Adverse Events (AEs) were collected from the signature of the Informed Consent Form through the follow-up period (30-37 days after the last visit, which was Week 312).
Reported AEs are treatment-emergent that is AEs that developed/worsened during the 'on treatment period' (first dose of eliglustat to end of follow-up period).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER