Trial Outcomes & Findings for A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris (NCT NCT02704429)
NCT ID: NCT02704429
Last Updated: 2023-02-13
Results Overview
Treatment-emergent adverse events (TEAEs) including clinically significant changes in physical examination, laboratory tests, and vital signs. An AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. TEAEs were defined as AEs that developed or worsened or became serious during on-treatment phase that was defined as the time from the start of study drug up to study completion.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
42 participants
Primary outcome timeframe
Part A: until 24 weeks and Part B: until 28 weeks
Results posted on
2023-02-13
Participant Flow
The study was conducted at 13 centers in 5 countries from 22 January 2016 to 10 January 2020.
A total of 69 patients were screened for the study (52 patients in Part A and 18\* patients in Part B). Of these, 41 unique patients were enrolled in the study (27 patients in Part A and 15\* patients in Part B). \*One patient who completed Part A of the study and later relapsed was enrolled in Part B.
Participant milestones
| Measure |
Part A
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Part A: 24 Weeks
STARTED
|
27
|
0
|
|
Part A: 24 Weeks
COMPLETED
|
24
|
0
|
|
Part A: 24 Weeks
NOT COMPLETED
|
3
|
0
|
|
Part B: 28 Weeks
STARTED
|
0
|
15
|
|
Part B: 28 Weeks
COMPLETED
|
0
|
14
|
|
Part B: 28 Weeks
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Part A
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Part A: 24 Weeks
Adverse Event
|
3
|
0
|
|
Part B: 28 Weeks
Worsening of pemphigus
|
0
|
1
|
Baseline Characteristics
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
Baseline characteristics by cohort
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
Total
n=42 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
Part A · <=18 years
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Age, Categorical
Part A · Between 18 and 65 years
|
24 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
24 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Age, Categorical
Part A · >=65 years
|
3 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
3 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Age, Categorical
Part B · <=18 years
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Age, Categorical
Part B · Between 18 and 65 years
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
15 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
15 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Age, Categorical
Part B · >=65 years
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Sex: Female, Male
Part A · Female
|
15 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
15 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Sex: Female, Male
Part A · Male
|
12 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
12 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Sex: Female, Male
Part B · Female
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
7 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
7 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Sex: Female, Male
Part B · Male
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
8 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
8 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · American Indian or Alaska Native
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · Asian
|
1 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
1 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · Black or African American
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · White
|
22 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
22 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · More than one race
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part A · Unknown or Not Reported
|
4 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
4 Participants
n=27 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · American Indian or Alaska Native
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · Asian
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
2 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
2 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · Native Hawaiian or Other Pacific Islander
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · Black or African American
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · White
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
8 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
8 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · More than one race
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
0 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
|
Race (NIH/OMB)
Part B · Unknown or Not Reported
|
0 Participants
One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
5 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
5 Participants
n=15 Participants • One patient who completed Part A of the study and later relapsed was enrolled in Part B.
|
PRIMARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: Safety Analysis population: All patients who received at least 1 dose of rilzabrutinib; used for all safety analyses
Treatment-emergent adverse events (TEAEs) including clinically significant changes in physical examination, laboratory tests, and vital signs. An AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. TEAEs were defined as AEs that developed or worsened or became serious during on-treatment phase that was defined as the time from the start of study drug up to study completion.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage of Participants With Treatment-emergent Adverse Events
Any TEAE
|
74.1 percentage of participants
|
86.7 percentage of participants
|
|
Percentage of Participants With Treatment-emergent Adverse Events
Any serious TEAE
|
11.1 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Treatment-emergent Adverse Events
Any TEAE leading to treatment discontinuation
|
11.1 percentage of participants
|
0 percentage of participants
|
PRIMARY outcome
Timeframe: 4 weeksPopulation: Intent-to-Treat (ITT) population: All patients who received at least 1 dose of rilzabrutinib
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage of Participants Who Are Able to Achieve Control of Disease Activity (CDA) Within 4 Weeks of Starting PRN1008 Treatment Without the Need for Doses of Prednisone or Prednisolone >0.5 mg/kg
|
51.9 percentage of participants
Interval 31.9 to 71.3
|
60.0 percentage of participants
Interval 32.29 to 83.66
|
SECONDARY outcome
Timeframe: 4 weeksPopulation: ITT
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage of Participants Able to Achieve Control of Disease Activity (CDA) Without Corticosteroids Within 4 Weeks
|
11.1 percentage of participants
Interval 2.4 to 29.2
|
6.7 percentage of participants
Interval 0.17 to 31.95
|
SECONDARY outcome
Timeframe: Part A: 12 weeks treatment and Part B: 24 weeks treatmentPopulation: ITT
CR was defined as complete healing of all lesions and the absence of new lesions.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage of Participants Able to Achieve a Complete Response (CR) Without Corticosteroids
|
0 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Part A: 12 weeks treatment and Part B: 24 weeks treatmentPopulation: ITT
CR was defined as complete healing of all lesions and the absence of new lesions.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage of Participants Able to Achieve Complete Remission (CR) Without the Need for Doses of Prednisone or Prednisolone of Greater Than 0.5mg/kg
|
14.8 percentage of participants
Interval 4.2 to 33.7
|
33.3 percentage of participants
Interval 11.82 to 61.62
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal. Kaplan-Meier estimate median time is reported.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Time to Control of Disease Activity (CDA)
|
33.0 days
Interval 29.0 to 58.0
|
29.0 days
Interval 15.0 to 34.0
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
ECP was defined as the time at which no new lesions have developed for minimum of 2 weeks, and approximately 80% of existing lesions have healed. The median time to ECP was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Time to End of Consolidation Phase (ECP)
|
170.0 days
Interval 95.0 to
The upper CI was not estimable due to the insufficient number of patients with an ECP response.
|
58.0 days
Interval 48.0 to
The upper CI was not estimable due to the insufficient number of patients with an ECP response.
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
CR was defined as complete healing of all lesions and the absence of new lesions.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Time to Complete Remission (CR)
|
NA days
Less than 50% patients achieved CR.
|
NA days
Less than 50% patients achieved CR.
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
Relapse was defined as appearance of ≥3 new lesions/month that do not heal spontaneously within 1 week, or by extension of established lesions, in a patient who has achieved disease control. The median time to relapse was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Time to Relapse After PRN1008 Treatment Discontinuation
|
96.0 days
Interval 27.0 to 99.0
|
198.0 days
Interval 41.0 to 209.0
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
Cumulative corticosteroid usage
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Cumulative Corticosteroid Usage
|
983.43 mg
Standard Deviation 827.676
|
2089.600 mg
Standard Deviation 1274.011
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
The PDAI questionnaire has 2 components including activity and damage. The activity component consists of skin, scalp and mucosa parts, and the damage component consists of skin and scalp parts. The total activity score was used for the summary of PDAI scores. PDAI total activity score = Total skin activity + Total scalp activity + Total mucosa activity. PDAI Total Activity Score ranged from 0 to 250 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Percentage Change From Baseline in Pemphigus Disease Area Index (PDAI) Total Activity Scores
end of treatment
|
-55.7 score on a scale
Standard Deviation 40.91
|
-78.60 score on a scale
Standard Deviation 35.839
|
|
Percentage Change From Baseline in Pemphigus Disease Area Index (PDAI) Total Activity Scores
end of follow-up
|
-57.7 score on a scale
Standard Deviation 38.45
|
-59.68 score on a scale
Standard Deviation 48.403
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
ABSIS Total Activity Score ranged from 0 to 206 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Change From Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) Total Activity Score
end of treatment
|
-8.18 score on a scale
Standard Deviation 14.475
|
-6.591 score on a scale
Standard Deviation 4.7664
|
|
Change From Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) Total Activity Score
end of follow-up
|
-9.98 score on a scale
Standard Deviation 18.369
|
-5.705 score on a scale
Standard Deviation 4.6248
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
ABQOL score ranged from 0 to 68 points representing disease activity (higher scores mean a worse outcome).
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Change From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL)
end of treatment
|
-3.7 score on a scale
Standard Deviation 6.96
|
-6.36 score on a scale
Standard Deviation 9.394
|
|
Change From Baseline in Autoimmune Bullous Diseases Quality of Life (ABQOL)
end of follow-up
|
-2.9 score on a scale
Standard Deviation 6.71
|
-3.79 score on a scale
Standard Deviation 9.569
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
TABQOL score ranged from 0 to 68 points representing disease activity (higher scores mean a worse outcome).
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Change From Baseline in Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) Scores
end of treatment
|
-0.0 score on a scale
Standard Deviation 6.73
|
-2.00 score on a scale
Standard Deviation 6.051
|
|
Change From Baseline in Treatment of Autoimmune Bullous Diseases Quality of Life (TABQOL) Scores
end of follow-up
|
-0.1 score on a scale
Standard Deviation 5.92
|
-2.14 score on a scale
Standard Deviation 5.655
|
SECONDARY outcome
Timeframe: Part A: until 24 weeks and Part B: until 28 weeksPopulation: ITT
Simplified Nutritional Appetite Questionnaire (SNAQ) score ranged from 0 to 20 points representing disease activity (higher scores mean a better outcome).
Outcome measures
| Measure |
Part A
n=27 Participants
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 Participants
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Change From Baseline in Appetite (SNAQ Score)
end of treatment
|
1.1 units on a scale
Standard Deviation 2.45
|
0.21 units on a scale
Standard Deviation 2.082
|
|
Change From Baseline in Appetite (SNAQ Score)
end of follow-up
|
1.0 units on a scale
Standard Deviation 2.84
|
0.50 units on a scale
Standard Deviation 2.103
|
Adverse Events
Part A
Serious events: 3 serious events
Other events: 20 other events
Deaths: 0 deaths
Part B
Serious events: 0 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Part A
n=27 participants at risk
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 participants at risk
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Congenital, familial and genetic disorders
Pulmonary sequestration
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Pancreatic pseudocyst
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Cellulitis
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
Other adverse events
| Measure |
Part A
n=27 participants at risk
Open-label PRN1008, 12 weeks; 12 weeks follow-up
|
Part B
n=15 participants at risk
Open-label PRN1008, 24 weeks; 4 weeks follow-up
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
22.2%
6/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
26.7%
4/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
11.1%
3/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
3/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Dry mouth
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Vomiting
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Staphylococcal skin infection
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Upper respiratory tract infection
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Headache
|
14.8%
4/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Dizziness
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
13.3%
2/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Paraesthesia
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
General disorders
Fatigue
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
General disorders
Peripheral swelling
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Ear and labyrinth disorders
Vertigo
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Psychiatric disorders
Anxiety
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
0.00%
0/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
7.4%
2/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Abdominal distension
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
13.3%
2/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Change of bowel habit
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Stomatitis
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Teeth brittle
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Dermatophytosis
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Nasopharyngitis
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Tracheitis
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Infections and infestations
Vaginal infection
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Androgenetic alopecia
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Erythema nodosum
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Migraine
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Nervous system disorders
Sciatica
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Eye disorders
Conjunctival haemorrhage
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Eye disorders
Eye pain
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Eye disorders
Vision blurred
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Investigations
Activated partial thromboplastin time prolonged
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Investigations
Blood glucose increased
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Investigations
International normalised ratio increased
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
General disorders
Asthenia
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
General disorders
Oedema peripheral
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Ear and labyrinth disorders
Ear pain
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Hepatobiliary disorders
Non-alcoholic steatohepatitis
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Metabolism and nutrition disorders
Type 2 diabetes mellitus
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Anogenital warts
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Psychiatric disorders
Terminal insomnia
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Renal and urinary disorders
Oliguria
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Reproductive system and breast disorders
Amenorrhoea
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Vascular disorders
Hot flush
|
0.00%
0/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Haematemesis
|
3.7%
1/27 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
6.7%
1/15 • Part A: until 24 weeks and Part B: until 28 weeks
Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period. Analysis was performed on safety population.
|
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