Trial Outcomes & Findings for Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B (NCT NCT01505062)
NCT ID: NCT01505062
Last Updated: 2022-04-28
Results Overview
An adverse event (AE) was any unfavorable and unintended physical sign, symptom, or laboratory parameter that developed or worsened in severity during the course of the study, whether or not considered related to the IMP. The TEAEs were defined as any event that started or increased in severity after the participant received IMP, including abnormal laboratory results, electrocardiogram, etc.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
9 participants
Primary outcome timeframe
From Baseline to Week 48
Results posted on
2022-04-28
Participant Flow
In 2017, Sanofi suspended trials of SAR421869, while assessing its future. Until final decision was made, trial TDU13600 was not complete and in fact, further recruitment was planned. In 2019, Sanofi decided to terminate trial due to final decision on SAR421869, and shared decision with health authorities. Results have been reported expeditiously.
A total of 11 participants were screened. Nine participants of them were enrolled in the study with 3 participants in each of Cohorts 1 to 3. Due to early termination no participant was recruited in Cohorts 4 and 5.
Participant milestones
| Measure |
Cohort 1
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 transducing units (TU) per eye.
|
Cohort 2
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
|---|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
Baseline characteristics by cohort
| Measure |
Cohort 1
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 TU per eye.
|
Cohort 2
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
25 years
n=93 Participants
|
42 years
n=4 Participants
|
50 years
n=27 Participants
|
32 years
n=483 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
6 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
3 Participants
n=483 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
9 Participants
n=483 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
PRIMARY outcome
Timeframe: From Baseline to Week 48Population: Analysis was performed on safety population.
An adverse event (AE) was any unfavorable and unintended physical sign, symptom, or laboratory parameter that developed or worsened in severity during the course of the study, whether or not considered related to the IMP. The TEAEs were defined as any event that started or increased in severity after the participant received IMP, including abnormal laboratory results, electrocardiogram, etc.
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 TU per eye.
|
Cohort 2
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
|---|---|---|---|
|
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
|
100 percentage of participants
|
100 percentage of participants
|
100 percentage of participants
|
PRIMARY outcome
Timeframe: From Baseline to Week 48Population: Analysis was performed on safety population.
An AE was any unfavorable and unintended physical sign, symptom, or laboratory parameter that developed or worsened in severity during the course of the study, whether or not considered related to the IMP. The TEAEs were defined as any event that started or increased in severity after the participant received IMP, including abnormal laboratory results, electrocardiogram, etc. For each AE, the severity was categorized as either mild, moderate or severe where 'mild' was defined as discomfort noticed but did not interfere with the participant's daily routines (an annoyance), 'moderate' was defined as some impairment of function, not hazardous to health (uncomfortable or embarrassing), and 'severe' was defined as significant impairment of function, hazardous to health (incapacitating).
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 TU per eye.
|
Cohort 2
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
n=3 Participants
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
|---|---|---|---|
|
Percentage of Participants With TEAEs by Severity
Mild
|
100 percentage of participants
|
100 percentage of participants
|
100 percentage of participants
|
|
Percentage of Participants With TEAEs by Severity
Moderate
|
0 percentage of participants
|
33 percentage of participants
|
67 percentage of participants
|
|
Percentage of Participants With TEAEs by Severity
Severe
|
0 percentage of participants
|
0 percentage of participants
|
67 percentage of participants
|
Adverse Events
Cohort 1
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 2
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 3
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 TU per eye.
|
Cohort 2
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
|---|---|---|---|
|
Eye disorders
Uveitis
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Visual acuity reduced
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
Other adverse events
| Measure |
Cohort 1
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^5 TU per eye.
|
Cohort 2
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 4.7\*10\^5 TU per eye.
|
Cohort 3
n=3 participants at risk
Participants received subretinally a single injection of SAR421869 at target dose of 1.4\*10\^6 TU per eye.
|
|---|---|---|---|
|
Eye disorders
Vitreous floaters
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Anterior chamber cell
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Cataract
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Conjunctival haemorrhage
|
100.0%
3/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Dyschromatopsia
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Eye pain
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Eye pruritus
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Macular fibrosis
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Photophobia
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Photopsia
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Retinal detachment
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Retinal haemorrhage
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Subretinal fluid
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Vision blurred
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Eye disorders
Visual acuity reduced
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Dry mouth
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
General disorders
Chest pain
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
General disorders
Fatigue
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
General disorders
Injection site extravasation
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Investigations
Blood creatine phosphokinase increased
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Investigations
Intraocular pressure decreased
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Investigations
Intraocular pressure increased
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
66.7%
2/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Investigations
Urine analysis abnormal
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Investigations
White blood cell count increased
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Metabolism and nutrition disorders
Increased appetite
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Nervous system disorders
Migraine
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Nervous system disorders
Occipital neuralgia
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Nervous system disorders
Syncope
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Psychiatric disorders
Nervousness
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Renal and urinary disorders
Glycosuria
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Renal and urinary disorders
Pollakiuria
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
|
Renal and urinary disorders
Urge incontinence
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
0.00%
0/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
33.3%
1/3 • All AEs were collected from time of first dose of study drug up to Week 48 regardless of seriousness or relationship (causality) to investigational product.
Reported AEs were TEAEs that developed/worsened during the 'on treatment period' (from Day 0 to Week 48). Analysis was performed on safety population.
|
Additional Information
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