Trial Outcomes & Findings for Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada (NCT NCT01557400)
NCT ID: NCT01557400
Last Updated: 2020-11-25
Results Overview
A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
94 participants
Primary outcome timeframe
Baseline up to Week 246
Results posted on
2020-11-25
Participant Flow
The treatment gap between the date of administration of the last dose of ataluren in Study PTC124-GD-007-DMD (NCT00592553) and Study PTC124-GD-007e-DMD (NCT00847379) and the date of administration of the first dose of ataluren in this study (PTC124-GD-019-DMD) ranged from 114.43 to 266.14 weeks (801 to 1863 days).
Of the 94 enrolled participants, 44 were not ambulatory and 84 were on concomitant therapy with corticosteroids. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
Participant milestones
| Measure |
Ataluren
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Overall Study
STARTED
|
94
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
94
|
|
Overall Study
COMPLETED
|
37
|
|
Overall Study
NOT COMPLETED
|
57
|
Reasons for withdrawal
| Measure |
Ataluren
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
|
Overall Study
Transitioned to Commercial Drug Product
|
40
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Lost to Follow-up
|
5
|
|
Overall Study
Withdrawal by Subject
|
9
|
Baseline Characteristics
All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable 6MWT data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
Baseline characteristics by cohort
| Measure |
Ataluren
n=94 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Age, Continuous
|
12.8 years
STANDARD_DEVIATION 2.38 • n=94 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=94 Participants
|
|
Sex: Female, Male
Male
|
94 Participants
n=94 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
87 participants
n=94 Participants
|
|
Race/Ethnicity, Customized
Asian
|
4 participants
n=94 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 participants
n=94 Participants
|
|
Race/Ethnicity, Customized
Unknown/Not Reported
|
1 participants
n=94 Participants
|
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6-Minute Walk Distance (6MWD) as Measured by the 6-Minute Walk Test (6MWT)
|
341.63 meters
STANDARD_DEVIATION 108.106 • n=49 Participants • All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable 6MWT data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
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|
Physical Function as Measured by the North Star Ambulatory Assessment (NSAA)
|
19.1 units on a scale
STANDARD_DEVIATION 8.52 • n=50 Participants • All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable NSAA data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
|
|
Time to Stand From Supine Position
|
18.56 seconds
STANDARD_DEVIATION 34.349 • n=38 Participants • All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable time to stand from supine data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
|
|
Time to Walk/Run 10 Meters
|
8.35 seconds
STANDARD_DEVIATION 4.693 • n=50 Participants • All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable data for time to walk/run 10 meters. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
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|
Pulmonary Function as Measured by Spirometry
FVC
|
1.94 liters
STANDARD_DEVIATION 0.509 • n=39 Participants • All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
|
|
Pulmonary Function as Measured by Spirometry
FEV1
|
68.60 liters
STANDARD_DEVIATION 18.295 • n=35 Participants • All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
|
|
Pulmonary Function as Measured by Spirometry
PEF
|
9.09 liters
STANDARD_DEVIATION 34.350 • n=39 Participants • All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
|
|
Pulmonary Function as Measured by Spirometry
PCF
|
33.06 liters
STANDARD_DEVIATION 81.985 • n=25 Participants • All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
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|
Participant and Parent/Caregiver-Reported Activities of Daily Living (ADL), as Measured by EK Scale
|
7.8 units on a scale
STANDARD_DEVIATION 3.76 • n=40 Participants • All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable EK scale data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
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PRIMARY outcome
Timeframe: Baseline up to Week 246Population: All enrolled participants who received at least 1 dose of study drug.
A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Ataluren
n=94 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Number of Participants With Treatment Emergent Adverse Events (TEAEs)
TEAEs
|
91 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Treatment-Emergent SAEs
|
31 Participants
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|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
TEAEs Related to Study Treatment
|
26 Participants
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SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable 6MWT data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
The 6MWD was assessed in participants who were ambulatory using standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Outcome measures
| Measure |
Ataluren
n=50 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Change From Baseline in 6MWD as Measured by the 6MWT
Week 48
|
-41.71 change in meters
Standard Deviation 48.172
|
|
Change From Baseline in 6MWD as Measured by the 6MWT
Week 96
|
-76.80 change in meters
Standard Deviation 70.781
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|
Change From Baseline in 6MWD as Measured by the 6MWT
Week 144
|
-97.57 change in meters
Standard Deviation 83.761
|
|
Change From Baseline in 6MWD as Measured by the 6MWT
Week 192
|
-109.37 change in meters
Standard Deviation 96.238
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Change From Baseline in 6MWD as Measured by the 6MWT
Week 240
|
-134.16 change in meters
Standard Deviation 94.716
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SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable NSAA data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
The NSAA was used to evaluate physical function in participants who were ambulatory at study entry, using standardized procedures. The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score. If fewer than 13 of the 17 activities were performed, the total score was considered missing. If 13 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. The linear score was the linear transformation of the NSAA score to a scale of 0 (worst) to 100 (best).
Outcome measures
| Measure |
Ataluren
n=50 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Change From Baseline in Physical Function as Measured by the NSAA
Week 48
|
-2.9 units on a scale
Standard Deviation 3.08
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|
Change From Baseline in Physical Function as Measured by the NSAA
Week 96
|
-7.4 units on a scale
Standard Deviation 5.61
|
|
Change From Baseline in Physical Function as Measured by the NSAA
Week 144
|
-8.8 units on a scale
Standard Deviation 6.29
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|
Change From Baseline in Physical Function as Measured by the NSAA
Week 240
|
-13.4 units on a scale
Standard Deviation 7.03
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SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable time to stand from supine data. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
Time to stand from the supine position to a standing position was assessed in ambulatory participants. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used.
Outcome measures
| Measure |
Ataluren
n=50 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Change From Baseline in Time to Stand From Supine Position
Week 48
|
3.33 seconds
Standard Deviation 5.390
|
|
Change From Baseline in Time to Stand From Supine Position
Week 96
|
6.38 seconds
Standard Deviation 6.029
|
|
Change From Baseline in Time to Stand From Supine Position
Week 144
|
6.11 seconds
Standard Deviation 6.798
|
|
Change From Baseline in Time to Stand From Supine Position
Week 192
|
3.84 seconds
Standard Deviation 8.410
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|
Change From Baseline in Time to Stand From Supine Position
Week 240
|
11.16 seconds
Standard Deviation 10.718
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SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable data for time to walk/run 10 meters. Participants who were ambulatory were able to run/walk 10 meters in ≤30 seconds at study entry.
Time to walk/run 10 meters was measured in ambulatory participants. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used.
Outcome measures
| Measure |
Ataluren
n=50 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Change From Baseline in Time to Walk/Run 10 Meters
Week 48
|
1.67 seconds
Standard Deviation 1.957
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|
Change From Baseline in Time to Walk/Run 10 Meters
Week 96
|
3.48 seconds
Standard Deviation 4.481
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|
Change From Baseline in Time to Walk/Run 10 Meters
Week 144
|
3.07 seconds
Standard Deviation 2.092
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Change From Baseline in Time to Walk/Run 10 Meters
Week 192
|
3.19 seconds
Standard Deviation 2.049
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Change From Baseline in Time to Walk/Run 10 Meters
Week 240
|
3.62 seconds
Standard Deviation 1.858
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SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable spirometry data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
Pulmonary function parameters of %-predicated FVC, percent-predicted FEV1 (adjusted using ulna length and age), PEF, and PCF was assessed in non-ambulatory participants by using a spirometer. Due to the difficulty in obtaining an accurate standing height measurement in non-ambulatory participants, ulna length and arm span were used as a surrogate measure for height when calculating percent-predicted FVC.
Outcome measures
| Measure |
Ataluren
n=43 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
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|---|---|
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Change From Baseline in Pulmonary Function as Measured by Spirometry
FVC, Week 48
|
-0.00 liters
Standard Deviation 0.239
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FVC, Week 96
|
-0.06 liters
Standard Deviation 0.391
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FVC, Week 144
|
-0.18 liters
Standard Deviation 0.422
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FVC, Week 192
|
-0.18 liters
Standard Deviation 0.760
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FVC, Week 240
|
-0.24 liters
Standard Deviation 0.720
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FEV1, Week 48
|
-7.98 liters
Standard Deviation 10.297
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FEV1, Week 96
|
-11.59 liters
Standard Deviation 13.366
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FEV1, Week 144
|
-20.60 liters
Standard Deviation 18.201
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FEV1, Week 192
|
-19.72 liters
Standard Deviation 21.916
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
FEV1, Week 240
|
-29.17 liters
Standard Deviation 18.759
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PEF, Week 48
|
-7.47 liters
Standard Deviation 40.552
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PEF, Week 96
|
9.67 liters
Standard Deviation 78.591
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PEF, Week 144
|
-8.34 liters
Standard Deviation 42.763
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PEF, Week 192
|
-9.66 liters
Standard Deviation 45.356
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PEF, Week 240
|
-23.77 liters
Standard Deviation 70.864
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PCF, Week 48
|
-3.67 liters
Standard Deviation 63.604
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PCF, Week 96
|
-14.34 liters
Standard Deviation 94.453
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PCF, Week 144
|
-48.51 liters
Standard Deviation 102.197
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PCF, Week 192
|
-52.19 liters
Standard Deviation 105.178
|
|
Change From Baseline in Pulmonary Function as Measured by Spirometry
PCF, Week 240
|
-81.38 liters
Standard Deviation 123.517
|
SECONDARY outcome
Timeframe: Baseline, Weeks 48, 96, 144, 192, and 240Population: All enrolled participants who received at least 1 dose of study drug, were non-ambulatory, and had evaluable EK scale data. Participants who were non-ambulatory were not able to run/walk 10 meters in ≤30 seconds at study entry.
Activities of daily living after loss of ambulation were measured using the EK scale. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move arms, 6) ability to use hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performs the tasks of daily life (as described by Categories 1 to 9) and how he perceives his wellbeing (as described by Category 10). The interviewer observed the participant and assigned the final score for the tasks that could be observed in the clinic.
Outcome measures
| Measure |
Ataluren
n=43 Participants
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
|
|---|---|
|
Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale
Week 48
|
2.0 units on a scale
Standard Deviation 3.03
|
|
Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale
Week 96
|
2.6 units on a scale
Standard Deviation 2.85
|
|
Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale
Week 144
|
3.3 units on a scale
Standard Deviation 2.31
|
|
Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale
Week 192
|
5.3 units on a scale
Standard Deviation 2.51
|
|
Change From Baseline in Participant and Parent/Caregiver-Reported ADL, as Measured by the EK Scale
Week 240
|
7.0 units on a scale
Standard Deviation 2.49
|
Adverse Events
Ataluren
Serious events: 31 serious events
Other events: 88 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Ataluren
n=94 participants at risk
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
|
|---|---|
|
Injury, poisoning and procedural complications
Femur fracture
|
16.0%
15/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Back injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Fall
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Pneumonia
|
3.2%
3/94 • Baseline up to Week 246
|
|
Infections and infestations
Actinomycosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Gastroenteritis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Postoperative abscess
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Urinary tract infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Ventricular arrhythmia
|
2.1%
2/94 • Baseline up to Week 246
|
|
Cardiac disorders
Cardiac failure
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Cardiogenic shock
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Myocardial infarction
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Tachycardia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
1.1%
1/94 • Baseline up to Week 246
|
|
Nervous system disorders
Intracranial pressure increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
1.1%
1/94 • Baseline up to Week 246
|
Other adverse events
| Measure |
Ataluren
n=94 participants at risk
Ataluren was provided as a vanilla-flavored powder to be mixed with water, milk, fruit juice (except apple juice) fruit punch, or in semi-solid food (for example, yogurt, pudding, or applesauce). The dose level for ataluren was 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug dosing was based on milligrams of drug per kilogram of body weight. Because of potential changes in participant body weight over time, weight-based dose adjustment occurred every 24 weeks as required. Study drug was taken for up to 240 weeks.
|
|---|---|
|
Investigations
Gamma-glutamyltransferase increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
High density lipoprotein increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Lymph node palpable
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Oxygen saturation decreased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Protein urine present
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Urinary lipids present
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Vomiting
|
29.8%
28/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Abdominal pain upper
|
14.9%
14/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Diarrhoea
|
13.8%
13/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Constipation
|
9.6%
9/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Nausea
|
7.4%
7/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Abdominal pain
|
6.4%
6/94 • Baseline up to Week 246
|
|
Infections and infestations
Nasopharyngitis
|
42.6%
40/94 • Baseline up to Week 246
|
|
Infections and infestations
Gastroenteritis
|
20.2%
19/94 • Baseline up to Week 246
|
|
Infections and infestations
Upper respiratory tract infection
|
20.2%
19/94 • Baseline up to Week 246
|
|
Infections and infestations
Influenza
|
9.6%
9/94 • Baseline up to Week 246
|
|
Infections and infestations
Rhinitis
|
9.6%
9/94 • Baseline up to Week 246
|
|
Infections and infestations
Lower respiratory tract infection
|
8.5%
8/94 • Baseline up to Week 246
|
|
Infections and infestations
Ear infection
|
7.4%
7/94 • Baseline up to Week 246
|
|
Infections and infestations
Respiratory tract infection
|
5.3%
5/94 • Baseline up to Week 246
|
|
Infections and infestations
Urinary tract infection
|
5.3%
5/94 • Baseline up to Week 246
|
|
General disorders
Disease progression
|
28.7%
27/94 • Baseline up to Week 246
|
|
General disorders
Pyrexia
|
20.2%
19/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Fall
|
22.3%
21/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Femur fracture
|
3.2%
3/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
7.4%
7/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Joint injury
|
5.3%
5/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.3%
5/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
22.3%
21/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.6%
9/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
9.6%
9/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
7.4%
7/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.4%
7/94 • Baseline up to Week 246
|
|
Nervous system disorders
Headache
|
30.9%
29/94 • Baseline up to Week 246
|
|
Nervous system disorders
Dizziness
|
6.4%
6/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
17.0%
16/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
14.9%
14/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
5/94 • Baseline up to Week 246
|
|
Cardiac disorders
Cardiomyopathy
|
8.5%
8/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.5%
8/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.3%
5/94 • Baseline up to Week 246
|
|
Cardiac disorders
Left ventricular dysfunction
|
3.2%
3/94 • Baseline up to Week 246
|
|
Cardiac disorders
Cardiovascular insufficiency
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Extrasystoles
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Left ventricular hypertrophy
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Sinus tachycardia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Cardiac disorders
Tachycardia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Abdominal discomfort
|
3.2%
3/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Flatulence
|
3.2%
3/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Dyspepsia
|
2.1%
2/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Haemorrhoids
|
2.1%
2/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Mouth ulceration
|
2.1%
2/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Toothache
|
2.1%
2/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Anal fistula
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Eructation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Faeces soft
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Frequent bowel movements
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Lip swelling
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Odynophagia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
1.1%
1/94 • Baseline up to Week 246
|
|
General disorders
Asthenia
|
4.3%
4/94 • Baseline up to Week 246
|
|
General disorders
Influenza like illness
|
4.3%
4/94 • Baseline up to Week 246
|
|
General disorders
Malaise
|
4.3%
4/94 • Baseline up to Week 246
|
|
General disorders
Fatigue
|
3.2%
3/94 • Baseline up to Week 246
|
|
General disorders
Chest pain
|
2.1%
2/94 • Baseline up to Week 246
|
|
General disorders
Infusion site pain
|
1.1%
1/94 • Baseline up to Week 246
|
|
General disorders
Non-cardiac chest pain
|
1.1%
1/94 • Baseline up to Week 246
|
|
General disorders
Oedema peripheral
|
1.1%
1/94 • Baseline up to Week 246
|
|
General disorders
Pain
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Pharyngitis
|
4.3%
4/94 • Baseline up to Week 246
|
|
Infections and infestations
Fungal infection
|
2.1%
2/94 • Baseline up to Week 246
|
|
Infections and infestations
Fungal skin infection
|
2.1%
2/94 • Baseline up to Week 246
|
|
Infections and infestations
Onychomycosis
|
2.1%
2/94 • Baseline up to Week 246
|
|
Infections and infestations
Paronychia
|
2.1%
2/94 • Baseline up to Week 246
|
|
Infections and infestations
Viral infection
|
2.1%
2/94 • Baseline up to Week 246
|
|
Infections and infestations
Actinomycosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Body tinea
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Bronchitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Cellulitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Conjunctivitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Gastroenteritis viral
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Gastrointestinal infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Gastrointestinal viral infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Herpes zoster
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Labyrinthitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Laryngitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Localised infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Pertussis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Pharyngitis streptococcal
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Pneumonia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Scarlet fever
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Sebaceous gland infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Subcutaneous abscess
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Tinea pedis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Tooth infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Viral pharyngitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Infections and infestations
Viral upper respiratory tract infection
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Foot fracture
|
3.2%
3/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Limb injury
|
3.2%
3/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Lumbar vertebral fracture
|
3.2%
3/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Contusion
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Muscle strain
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
2.1%
2/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Administration related reaction
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Back injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Bone fissure
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Chest injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Chillblains
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Eschar
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Hand fracture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Head injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Muscle rupture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Spinal column injury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Spinal cord injury sacral
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Dehydration
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Obesity
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Overweight
|
1.1%
1/94 • Baseline up to Week 246
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Extremity contracture
|
3.2%
3/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
3.2%
3/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
3.2%
3/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Fracture pain
|
2.1%
2/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Muscle contracture
|
2.1%
2/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Spinal deformity
|
2.1%
2/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Coccydynia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc compression
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Joint ankylosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Kyphosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Limb discomfort
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Lordosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Mastication disorder
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal discomfort
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Neck mass
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Tendon disorder
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Musculoskeletal and connective tissue disorders
Torticollis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
1.1%
1/94 • Baseline up to Week 246
|
|
Nervous system disorders
Migraine
|
4.3%
4/94 • Baseline up to Week 246
|
|
Nervous system disorders
Aphonia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Nervous system disorders
Hypokinesia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Nervous system disorders
Presyncope
|
1.1%
1/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Anxiety
|
3.2%
3/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Enuresis
|
3.2%
3/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Sleep disorder
|
2.1%
2/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Anger
|
1.1%
1/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Gender dysphoria
|
1.1%
1/94 • Baseline up to Week 246
|
|
Psychiatric disorders
Obsessive-compulsive disorder
|
1.1%
1/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Proteinuria
|
3.2%
3/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Dysuria
|
2.1%
2/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Urine abnormality
|
2.1%
2/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Incontinence
|
1.1%
1/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Myoglobinuria
|
1.1%
1/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Pollakiuria
|
1.1%
1/94 • Baseline up to Week 246
|
|
Renal and urinary disorders
Strangury
|
1.1%
1/94 • Baseline up to Week 246
|
|
Reproductive system and breast disorders
Testicular appendage torsion
|
1.1%
1/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
3.2%
3/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
3.2%
3/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
3.2%
3/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnoea syndrome
|
2.1%
2/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Hypoventilation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
1.1%
1/94 • Baseline up to Week 246
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary congestion
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Eczema
|
3.2%
3/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
2.1%
2/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
2.1%
2/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
2.1%
2/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Intertrigo
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Pain of skin
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Pityriasis rosea
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Skin fissures
|
1.1%
1/94 • Baseline up to Week 246
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
1.1%
1/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Tenotomy
|
3.2%
3/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Haemorrhoid operation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Nail operation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Spinal operation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Tooth extraction
|
1.1%
1/94 • Baseline up to Week 246
|
|
Surgical and medical procedures
Wedge resection toenail
|
1.1%
1/94 • Baseline up to Week 246
|
|
Vascular disorders
Hypertension
|
1.1%
1/94 • Baseline up to Week 246
|
|
Vascular disorders
Hypotension
|
1.1%
1/94 • Baseline up to Week 246
|
|
Vascular disorders
Varicose ulceration
|
1.1%
1/94 • Baseline up to Week 246
|
|
Ear and labyrinth disorders
Vertigo
|
3.2%
3/94 • Baseline up to Week 246
|
|
Ear and labyrinth disorders
Ear pain
|
2.1%
2/94 • Baseline up to Week 246
|
|
Ear and labyrinth disorders
Motion sickness
|
2.1%
2/94 • Baseline up to Week 246
|
|
Endocrine disorders
Delayed puberty
|
2.1%
2/94 • Baseline up to Week 246
|
|
Eye disorders
Cataract
|
4.3%
4/94 • Baseline up to Week 246
|
|
Eye disorders
Eye allergy
|
1.1%
1/94 • Baseline up to Week 246
|
|
Eye disorders
Myopia
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Cardiac function test abnormal
|
3.2%
3/94 • Baseline up to Week 246
|
|
Investigations
Cortisol decreased
|
3.2%
3/94 • Baseline up to Week 246
|
|
Investigations
Weight decreased
|
3.2%
3/94 • Baseline up to Week 246
|
|
Investigations
Weight increased
|
3.2%
3/94 • Baseline up to Week 246
|
|
Investigations
Cystatin C increased
|
2.1%
2/94 • Baseline up to Week 246
|
|
Investigations
Monocyte count decreased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Red blood cells urine
|
2.1%
2/94 • Baseline up to Week 246
|
|
Investigations
Blood bilirubin increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood cholesterol increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood corticotrophin decreased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood lactate dehydrogenase increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood magnesium increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood potassium increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood sodium decreased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood testosterone decreased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood urea increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood uric acid increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Blood urine present
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Body temperature increased
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Electrocardiogram ST segment elevation
|
1.1%
1/94 • Baseline up to Week 246
|
|
Investigations
Forced vital capacity decreased
|
1.1%
1/94 • Baseline up to Week 246
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER