The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (ACT DMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01826487
Recruitment Status : Completed
First Posted : April 8, 2013
Results First Posted : August 4, 2020
Last Update Posted : August 4, 2020
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Interventions Drug: Ataluren
Drug: Placebo
Enrollment 230
Recruitment Details A total of 291 participants were screened for eligibility, of which 61 participants did not meet entry criteria.
Pre-assignment Details A total of 230 eligible participants were randomized in 1:1 ratio to receive either placebo or ataluren. 2 participants, 1 in each treatment arm, were excluded from intent-to-treat (ITT) population; as they did not have at least 1 valid post-baseline 6-minute walk distance (6MWD) value, a requirement for inclusion in ITT population.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description Participants received placebo matched to ataluren orally 3 times a day (TID) at morning, midday, and evening for 48 weeks. Participants received ataluren suspension orally TID, 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Period Title: Overall Study
Started 115 115
As-treated Population [1] 115 115
ITT Population [2] 114 114
Completed 111 110
Not Completed 4 5
Reason Not Completed
Adverse Event             1             1
Lost to Follow-up             1             0
Withdrawal by Subject             1             3
Protocol Violation             1             1
[1]
All Randomized participants who received any study treatment.
[2]
Received any study treatment; had a valid baseline, and at least 1 valid post-baseline 6MWD value.
Arm/Group Title Placebo Ataluren Total
Hide Arm/Group Description Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks. Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks. Total of all reporting groups
Overall Number of Baseline Participants 115 115 230
Hide Baseline Analysis Population Description
As-treated population included all randomized participants who actually received any study treatment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 115 participants 115 participants 230 participants
9.0  (1.65) 8.9  (1.79) 8.9  (1.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 115 participants 115 participants 230 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
115
 100.0%
115
 100.0%
230
 100.0%
6 Minute Walk Distance (6MWD)   [1] 
Mean (Standard Deviation)
Unit of measure:  Meters
Number Analyzed 115 participants 115 participants 230 participants
362.69  (81.424) 364.04  (73.342) 363.36  (77.322)
[1]
Measure Description: The 6-minute walk distance (6MWD) test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Time to Walk/Run 10 Meters  
Mean (Standard Deviation)
Unit of measure:  Seconds
Number Analyzed 115 participants 115 participants 230 participants
6.83  (2.924) 6.66  (3.078) 6.75  (2.996)
Time to Climb 4 Stairs   [1] 
Mean (Standard Deviation)
Unit of measure:  Seconds
Number Analyzed 112 participants 112 participants 224 participants
6.76  (7.287) 5.99  (5.347) 6.38  (6.389)
[1]
Measure Analysis Population Description: 'Number analyzed' signifies participants of as-treated population analyzed for this parameter.
Time to Descend 4 Stairs   [1] 
Mean (Standard Deviation)
Unit of measure:  Seconds
Number Analyzed 109 participants 112 participants 221 participants
5.05  (5.362) 5.07  (5.157) 5.06  (5.247)
[1]
Measure Analysis Population Description: 'Number analyzed' signifies participants of as-treated population analyzed for this parameter.
Physical Function Total Score as Measured by North Star Ambulatory Assessment (NSAA)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 114 participants 114 participants 228 participants
60.2  (18.37) 60.9  (17.97) 60.6  (18.14)
[1]
Measure Description: NSAA comprised tests of 17 abilities, such as ability to stand, rise from floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." Sum of these 17 scores was reported as ordinal total score, which can be transformed to a linear total score from 0 (worst) to 100 (best).
[2]
Measure Analysis Population Description: Intent-to-treat (ITT) population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value.
Baseline 6MWT   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
<300 meters Number Analyzed 115 participants 115 participants 230 participants
22
  19.1%
25
  21.7%
47
  20.4%
>=300 to <400 meters Number Analyzed 115 participants 115 participants 230 participants
52
  45.2%
47
  40.9%
99
  43.0%
>=400 meters Number Analyzed 115 participants 115 participants 230 participants
41
  35.7%
43
  37.4%
84
  36.5%
[1]
Measure Description: The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Pediatric Outcomes Data Collection Instrument (PODCI) Scores   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Transfers/Basic Mobility Score Number Analyzed 114 participants 114 participants 228 participants
81.4  (15.79) 83.9  (13.10) 82.7  (14.53)
Sports/Physical Functioning Score Number Analyzed 114 participants 114 participants 228 participants
56.0  (20.94) 56.2  (18.94) 56.1  (19.92)
[1]
Measure Description: PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity & Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. Following PODCI domains were prespecified in protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 (poor outcome/worse health) to 100(the highest level of functioning & least pain).
[2]
Measure Analysis Population Description: ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value.
1.Primary Outcome
Title Change From Baseline in 6MWD at Week 48
Hide Description The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline and Week 48 6MWD values are each the average of two valid 6MWD values, or a single available value if one was missing.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Multiple imputation was applied to impute missing values within the treatment groups.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 114 114
Least Squares Mean (Standard Error)
Unit of Measure: meters
-60.67  (9.323) -47.69  (9.247)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Ataluren
Comments Analysis was performed using analysis of covariance (ANCOVA) method including stratification factors for age (less than [<] 9 years versus [vs.] greater than or equal to [>=] 9 years), duration of use of corticosteroids at baseline (approx. >=6 to <12 months vs. >=12 months), and baseline 6MWD category (>=350 meters vs <350 meters), as well as baseline 6MWD as covariate.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.213
Comments Threshold for significance at 0.05. Secondary endpoints were tested for significance, only if the primary endpoint was statistically significant.
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 12.98
Confidence Interval (2-Sided) 95%
-7.44 to 33.39
Parameter Dispersion
Type: Standard Error of the Mean
Value: 10.415
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Time to 10 Percent (%) Persistent Worsening in 6MWD
Hide Description The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Time to 10% persistent worsening in 6MWD <300 meters, >=300 to 400 meters, and >=400 meters was evaluated. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of the last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening.
Time Frame Baseline to Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Multiple imputation was applied to impute missing values within treatment groups. Here, 'Number analyzed 'signifies participants evaluable for specified categories.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 114 114
Median (95% Confidence Interval)
Unit of Measure: days
<300 meters Number Analyzed 21 participants 24 participants
56
(1.0 to 111.0)
164
(1.0 to 225.0)
>=300 to <400 meters Number Analyzed 52 participants 47 participants
280 [1] 
(169.0 to NA)
NA [2] 
(280.0 to NA)
>=400 meters Number Analyzed 41 participants 43 participants
NA [3] 
(NA to NA)
NA [3] 
(NA to NA)
[1]
Due to smaller number of participants with an event, upper limit of 95% confidence interval (CI) could not be calculated.
[2]
Due to smaller number of participants with an event, median and upper limit of 95% CI could not be calculated.
[3]
Due to smaller number of participants with an event, median and 95% CI could not be calculated.
3.Secondary Outcome
Title Change From Baseline in Time to Walk/Run 10 Meters at Week 48
Hide Description During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses ] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). 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. A cumulative change from baseline data has been reported.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 110 109
Mean (Standard Deviation)
Unit of Measure: seconds
3.47  (6.393) 2.27  (5.216)
4.Secondary Outcome
Title Change From Baseline in Time to Climb 4 Stairs at Week 48
Hide Description During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 103 105
Mean (Standard Deviation)
Unit of Measure: seconds
4.46  (7.310) 2.65  (5.297)
5.Secondary Outcome
Title Change From Baseline in Time to Descend 4 Stairs at Week 48
Hide Description During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 100 106
Mean (Standard Deviation)
Unit of Measure: seconds
3.97  (7.854) 2.15  (5.306)
6.Secondary Outcome
Title Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
Hide Description An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an 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); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. Treatment-emergent adverse event (TEAE) was defined as an adverse event that occurred or worsened in the period extending from first dose of study drug to 6 weeks after the last dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Time Frame Baseline up to Week 54
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 115 115
Measure Type: Number
Unit of Measure: percentage of participants
87.8 89.6
7.Other Pre-specified Outcome
Title Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48
Hide Description Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 108 106
Mean (Standard Deviation)
Unit of Measure: units on a scale
-8.4  (10.65) -6.3  (10.64)
8.Other Pre-specified Outcome
Title Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel
Hide Description Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 48), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much worse), 2 (slightly worse), 3 (unchanged), 4 (slightly better), or 5 (much better).
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed 'signifies participants evaluable for specified categories.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 114 114
Measure Type: Count of Participants
Unit of Measure: Participants
Physical Functioning: Upper Extremity Activity Number Analyzed 82 participants 88 participants
Much better
1
   1.2%
4
   4.5%
Slightly better
7
   8.5%
7
   8.0%
Unchanged
67
  81.7%
73
  83.0%
Slightly worse
6
   7.3%
2
   2.3%
Much worse
1
   1.2%
2
   2.3%
Physical Functioning: Walking Number Analyzed 112 participants 108 participants
Much better
5
   4.5%
8
   7.4%
Slightly better
13
  11.6%
16
  14.8%
Unchanged
57
  50.9%
60
  55.6%
Slightly worse
19
  17.0%
21
  19.4%
Much worse
18
  16.1%
3
   2.8%
Physical Functioning: Climbing Stairs Number Analyzed 105 participants 110 participants
Much better
4
   3.8%
4
   3.6%
Slightly better
8
   7.6%
13
  11.8%
Unchanged
61
  58.1%
65
  59.1%
Slightly worse
17
  16.2%
15
  13.6%
Much worse
15
  14.3%
13
  11.8%
Physical Functioning: Other Number Analyzed 65 participants 68 participants
Much better
4
   6.2%
5
   7.4%
Slightly better
9
  13.8%
10
  14.7%
Unchanged
38
  58.5%
44
  64.7%
Slightly worse
7
  10.8%
8
  11.8%
Much worse
7
  10.8%
1
   1.5%
Cognition/Social Functioning Number Analyzed 92 participants 104 participants
Much better
6
   6.5%
5
   4.8%
Slightly better
12
  13.0%
20
  19.2%
Unchanged
71
  77.2%
74
  71.2%
Slightly worse
3
   3.3%
3
   2.9%
Much worse
0
   0.0%
2
   1.9%
Emotional/Social Functioning Number Analyzed 94 participants 105 participants
Much better
3
   3.2%
8
   7.6%
Slightly better
9
   9.6%
21
  20.0%
Unchanged
75
  79.8%
68
  64.8%
Slightly worse
5
   5.3%
6
   5.7%
Much worse
2
   2.1%
2
   1.9%
General Energy Level Number Analyzed 83 participants 89 participants
Much better
3
   3.6%
8
   9.0%
Slightly better
12
  14.5%
12
  13.5%
Unchanged
54
  65.1%
63
  70.8%
Slightly worse
11
  13.3%
5
   5.6%
Much worse
3
   3.6%
1
   1.1%
Sleep Number Analyzed 85 participants 91 participants
Much better
3
   3.5%
3
   3.3%
Slightly better
4
   4.7%
8
   8.8%
Unchanged
73
  85.9%
76
  83.5%
Slightly worse
5
   5.9%
2
   2.2%
Much worse
0
   0.0%
2
   2.2%
Other Number Analyzed 23 participants 23 participants
Much better
0
   0.0%
1
   4.3%
Slightly better
5
  21.7%
6
  26.1%
Unchanged
15
  65.2%
13
  56.5%
Slightly worse
2
   8.7%
3
  13.0%
Much worse
1
   4.3%
0
   0.0%
9.Other Pre-specified Outcome
Title Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48
Hide Description Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domains were prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.
Time Frame Baseline, Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 110 109
Mean (Standard Deviation)
Unit of Measure: units on a scale
Transfers/Basic Mobility Score -8.8  (15.80) -6.6  (14.76)
Sports/Physical Functioning Score -7.3  (15.87) -5.6  (15.91)
10.Other Pre-specified Outcome
Title Ataluren Plasma Concentration
Hide Description Plasma samples for the determination of ataluren concentrations were analyzed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation of 0.5 micrograms/milliliter (mcg/mL).
Time Frame Weeks 8, 16, 24, 32, 40, and 48
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all randomized participants who actually received any study treatment. Here, 'Number analyzed 'signifies participants evaluable at specified timepoint.
Arm/Group Title Ataluren
Hide Arm/Group Description:
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 115
Mean (Standard Deviation)
Unit of Measure: mcg/mL
Week 08 Number Analyzed 113 participants
4.230  (5.3913)
Week 16 Number Analyzed 113 participants
3.429  (3.9275)
Week 24 Number Analyzed 112 participants
3.323  (3.6135)
Week 32 Number Analyzed 113 participants
3.480  (3.1053)
Week 40 Number Analyzed 111 participants
3.997  (4.7615)
Week 48 Number Analyzed 110 participants
3.544  (3.8082)
11.Other Pre-specified Outcome
Title Study Drug Compliance
Hide Description Study drug compliance was assessed by quantification of used and unused study drug. Compliance was assessed in terms of the percentage of drug actually taken relative to the amount that should have been taken during the study.
Time Frame Baseline to Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description:
Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
Overall Number of Participants Analyzed 115 115
Mean (Standard Deviation)
Unit of Measure: percentage of drug
95.1  (9.43) 95.7  (7.57)
Time Frame Baseline up to 6 weeks after the last dose of study drug (Week 54)
Adverse Event Reporting Description As-treated population included all randomized participants who actually received any study treatment.
 
Arm/Group Title Placebo Ataluren
Hide Arm/Group Description Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks. Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.
All-Cause Mortality
Placebo Ataluren
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Placebo Ataluren
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/115 (3.48%)      4/115 (3.48%)    
Cardiac disorders     
Myocarditis † 1  0/115 (0.00%)  0 1/115 (0.87%)  1
Hepatobiliary disorders     
Hepatic function abnormal † 1  0/115 (0.00%)  0 1/115 (0.87%)  1
Infections and infestations     
Pneumonia † 1  2/115 (1.74%)  2 0/115 (0.00%)  0
Bronchiolitis † 1  1/115 (0.87%)  1 0/115 (0.00%)  0
Gastroenteritis † 1  0/115 (0.00%)  0 1/115 (0.87%)  1
Injury, poisoning and procedural complications     
Post-traumatic pain † 1  1/115 (0.87%)  1 0/115 (0.00%)  0
Femur fracture † 1  0/115 (0.00%)  0 1/115 (0.87%)  1
Lower limb fracture † 1  0/115 (0.00%)  0 1/115 (0.87%)  1
Musculoskeletal and connective tissue disorders     
Tendon disorder † 1  1/115 (0.87%)  1 0/115 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Adenoidal hypertrophy † 1  1/115 (0.87%)  1 0/115 (0.00%)  0
Nasal turbinate hypertrophy † 1  1/115 (0.87%)  1 0/115 (0.00%)  0
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA v15.1
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo Ataluren
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   99/115 (86.09%)      103/115 (89.57%)    
Blood and lymphatic system disorders     
Neutropenia † 1  0/115 (0.00%)  1/115 (0.87%) 
Splenomegaly † 1  0/115 (0.00%)  1/115 (0.87%) 
Leukocytosis † 1  1/115 (0.87%)  0/115 (0.00%) 
Lymphadenopathy † 1  1/115 (0.87%)  0/115 (0.00%) 
Cardiac disorders     
Right ventricular hypertrophy † 1  0/115 (0.00%)  2/115 (1.74%) 
Left ventricular dysfunction † 1  0/115 (0.00%)  1/115 (0.87%) 
Left ventricular hypertrophy † 1  0/115 (0.00%)  1/115 (0.87%) 
Myocardial fibrosis † 1  0/115 (0.00%)  1/115 (0.87%) 
Palpitations † 1  2/115 (1.74%)  1/115 (0.87%) 
Cardiomyopathy † 1  1/115 (0.87%)  0/115 (0.00%) 
Wolff-Parkinson-White syndrome † 1  1/115 (0.87%)  0/115 (0.00%) 
Ear and labyrinth disorders     
Ear pain † 1  4/115 (3.48%)  0/115 (0.00%) 
Middle ear effusion † 1  1/115 (0.87%)  0/115 (0.00%) 
Tympanic membrane disorder † 1  1/115 (0.87%)  0/115 (0.00%) 
Endocrine disorders     
Hypothyroidism † 1  0/115 (0.00%)  1/115 (0.87%) 
Hyperaldosteronism † 1  1/115 (0.87%)  0/115 (0.00%) 
Eye disorders     
Astigmatism † 1  0/115 (0.00%)  1/115 (0.87%) 
Cataract † 1  1/115 (0.87%)  1/115 (0.87%) 
Conjunctivitis † 1  2/115 (1.74%)  0/115 (0.00%) 
Eye allergy † 1  0/115 (0.00%)  1/115 (0.87%) 
Conjunctivitis allergic † 1  1/115 (0.87%)  0/115 (0.00%) 
Eye movement disorder † 1  1/115 (0.87%)  0/115 (0.00%) 
Eyelid oedema † 1  1/115 (0.87%)  0/115 (0.00%) 
Myopia † 1  1/115 (0.87%)  0/115 (0.00%) 
Gastrointestinal disorders     
Diarrhoea † 1  10/115 (8.70%)  20/115 (17.39%) 
Abdominal pain upper † 1  13/115 (11.30%)  9/115 (7.83%) 
Abdominal pain † 1  5/115 (4.35%)  7/115 (6.09%) 
Abdominal discomfort † 1  0/115 (0.00%)  3/115 (2.61%) 
Constipation † 1  10/115 (8.70%)  3/115 (2.61%) 
Flatulence † 1  1/115 (0.87%)  2/115 (1.74%) 
Abnormal faeces † 1  0/115 (0.00%)  1/115 (0.87%) 
Breath odour † 1  0/115 (0.00%)  1/115 (0.87%) 
Dental caries † 1  0/115 (0.00%)  1/115 (0.87%) 
Dry mouth † 1  1/115 (0.87%)  1/115 (0.87%) 
Dyspepsia † 1  3/115 (2.61%)  1/115 (0.87%) 
Gastritis † 1  1/115 (0.87%)  1/115 (0.87%) 
Gastrooesophageal reflux disease † 1  2/115 (1.74%)  1/115 (0.87%) 
Haemorrhoids † 1  1/115 (0.87%)  1/115 (0.87%) 
Anal fissure † 1  1/115 (0.87%)  0/115 (0.00%) 
Food poisoning † 1  1/115 (0.87%)  0/115 (0.00%) 
Haematochezia † 1  1/115 (0.87%)  0/115 (0.00%) 
Inguinal hernia † 1  1/115 (0.87%)  0/115 (0.00%) 
Irritable bowel syndrome † 1  1/115 (0.87%)  0/115 (0.00%) 
Vomiting † 1  21/115 (18.26%)  26/115 (22.61%) 
Nausea † 1  7/115 (6.09%)  7/115 (6.09%) 
Tooth crowding † 1  0/115 (0.00%)  2/115 (1.74%) 
Malocclusion † 1  0/115 (0.00%)  1/115 (0.87%) 
Odynophagia † 1  0/115 (0.00%)  1/115 (0.87%) 
Oral pain † 1  0/115 (0.00%)  1/115 (0.87%) 
Rectal haemorrhage † 1  2/115 (1.74%)  0/115 (0.00%) 
Swollen tongue † 1  1/115 (0.87%)  0/115 (0.00%) 
Toothache † 1  1/115 (0.87%)  0/115 (0.00%) 
General disorders     
Pyrexia † 1  12/115 (10.43%)  16/115 (13.91%) 
Disease progression † 1  14/115 (12.17%)  9/115 (7.83%) 
Abasia † 1  0/115 (0.00%)  3/115 (2.61%) 
Oedema peripheral † 1  0/115 (0.00%)  3/115 (2.61%) 
Fatigue † 1  3/115 (2.61%)  1/115 (0.87%) 
Non-cardiac chest pain † 1  1/115 (0.87%)  1/115 (0.87%) 
Influenza like illness † 1  2/115 (1.74%)  1/115 (0.87%) 
Gait disturbance † 1  1/115 (0.87%)  0/115 (0.00%) 
Malaise † 1  3/115 (2.61%)  0/115 (0.00%) 
Thirst † 1  1/115 (0.87%)  0/115 (0.00%) 
Hepatobiliary disorders     
Hepatic function abnormal † 1  1/115 (0.87%)  0/115 (0.00%) 
Hepatic steatosis † 1  1/115 (0.87%)  0/115 (0.00%) 
Immune system disorders     
Allergy to chemicals † 1  0/115 (0.00%)  1/115 (0.87%) 
Seasonal allergy † 1  2/115 (1.74%)  2/115 (1.74%) 
Rhinitis allergic † 1  0/115 (0.00%)  1/115 (0.87%) 
Allergy to vaccine † 1  1/115 (0.87%)  0/115 (0.00%) 
Drug hypersensitivity † 1  2/115 (1.74%)  0/115 (0.00%) 
Infections and infestations     
Ear infection † 1  1/115 (0.87%)  6/115 (5.22%) 
Gastroenteritis † 1  4/115 (3.48%)  5/115 (4.35%) 
Influenza † 1  5/115 (4.35%)  3/115 (2.61%) 
Bronchitis † 1  3/115 (2.61%)  2/115 (1.74%) 
Conjunctivitis infective † 1  0/115 (0.00%)  2/115 (1.74%) 
Fungal skin infection † 1  1/115 (0.87%)  2/115 (1.74%) 
Gastroenteritis viral † 1  0/115 (0.00%)  2/115 (1.74%) 
Gastrointestinal viral infection † 1  0/115 (0.00%)  2/115 (1.74%) 
Hordeolum † 1  3/115 (2.61%)  2/115 (1.74%) 
Eye infection † 1  1/115 (0.87%)  1/115 (0.87%) 
Gingivitis † 1  0/115 (0.00%)  1/115 (0.87%) 
Herpes simplex † 1  0/115 (0.00%)  1/115 (0.87%) 
Lower respiratory tract infection † 1  4/115 (3.48%)  1/115 (0.87%) 
Lung infection † 1  0/115 (0.00%)  1/115 (0.87%) 
Acute tonsillitis † 1  1/115 (0.87%)  0/115 (0.00%) 
Adenoiditis † 1  1/115 (0.87%)  0/115 (0.00%) 
Gastrointestinal infection † 1  2/115 (1.74%)  0/115 (0.00%) 
Gingival infection † 1  1/115 (0.87%)  0/115 (0.00%) 
Helminthic infection † 1  1/115 (0.87%)  0/115 (0.00%) 
Infected bites † 1  1/115 (0.87%)  0/115 (0.00%) 
Nasopharyngitis † 1  22/115 (19.13%)  24/115 (20.87%) 
Upper respiratory tract infection † 1  6/115 (5.22%)  11/115 (9.57%) 
Rhinitis † 1  4/115 (3.48%)  8/115 (6.96%) 
Pharyngitis † 1  4/115 (3.48%)  4/115 (3.48%) 
Tonsillitis † 1  2/115 (1.74%)  3/115 (2.61%) 
Sinusitis † 1  2/115 (1.74%)  5/115 (4.35%) 
Viral infection † 1  3/115 (2.61%)  3/115 (2.61%) 
Nail infection † 1  0/115 (0.00%)  2/115 (1.74%) 
Respiratory tract infection † 1  1/115 (0.87%)  2/115 (1.74%) 
Pneumonia † 1  0/115 (0.00%)  2/115 (1.74%) 
Otitis externa † 1  0/115 (0.00%)  1/115 (0.87%) 
Otitis media † 1  2/115 (1.74%)  1/115 (0.87%) 
Parasitic gastroenteritis † 1  0/115 (0.00%)  1/115 (0.87%) 
Skin infection † 1  1/115 (0.87%)  1/115 (0.87%) 
Mycoplasma infection † 1  1/115 (0.87%)  0/115 (0.00%) 
Oral candidiasis † 1  1/115 (0.87%)  0/115 (0.00%) 
Oral herpes † 1  1/115 (0.87%)  0/115 (0.00%) 
Pharyngitis streptococcal † 1  1/115 (0.87%)  0/115 (0.00%) 
Rubella † 1  1/115 (0.87%)  0/115 (0.00%) 
Tracheitis † 1  1/115 (0.87%)  0/115 (0.00%) 
Urinary tract infection † 1  2/115 (1.74%)  0/115 (0.00%) 
Injury, poisoning and procedural complications     
Arthropod bite † 1  0/115 (0.00%)  2/115 (1.74%) 
Arthropod sting † 1  1/115 (0.87%)  1/115 (0.87%) 
Concussion † 1  1/115 (0.87%)  0/115 (0.00%) 
Fall † 1  20/115 (17.39%)  21/115 (18.26%) 
Contusion † 1  4/115 (3.48%)  3/115 (2.61%) 
Ligament sprain † 1  7/115 (6.09%)  3/115 (2.61%) 
Laceration † 1  1/115 (0.87%)  2/115 (1.74%) 
Post-traumatic pain † 1  4/115 (3.48%)  2/115 (1.74%) 
Spinal compression fracture † 1  1/115 (0.87%)  2/115 (1.74%) 
Electrical burn † 1  0/115 (0.00%)  1/115 (0.87%) 
Excoriation † 1  1/115 (0.87%)  1/115 (0.87%) 
Fibula fracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Foot fracture † 1  1/115 (0.87%)  1/115 (0.87%) 
Hand fracture † 1  1/115 (0.87%)  1/115 (0.87%) 
Joint injury † 1  1/115 (0.87%)  1/115 (0.87%) 
Muscle injury † 1  0/115 (0.00%)  1/115 (0.87%) 
Muscle strain † 1  0/115 (0.00%)  1/115 (0.87%) 
Scratch † 1  0/115 (0.00%)  1/115 (0.87%) 
Tibia fracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Tooth injury † 1  0/115 (0.00%)  1/115 (0.87%) 
Ulna fracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Upper limb fracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Head injury † 1  1/115 (0.87%)  0/115 (0.00%) 
Limb injury † 1  1/115 (0.87%)  0/115 (0.00%) 
Lip injury † 1  1/115 (0.87%)  0/115 (0.00%) 
Thermal burn † 1  2/115 (1.74%)  0/115 (0.00%) 
Investigations     
Renin increased † 1  3/115 (2.61%)  1/115 (0.87%) 
Metabolism and nutrition disorders     
Hypertriglyceridaemia † 1  3/115 (2.61%)  5/115 (4.35%) 
Decreased appetite † 1  2/115 (1.74%)  3/115 (2.61%) 
Electrolyte imbalance † 1  1/115 (0.87%)  2/115 (1.74%) 
Vitamin D deficiency † 1  0/115 (0.00%)  2/115 (1.74%) 
Abnormal weight gain † 1  1/115 (0.87%)  1/115 (0.87%) 
Dyslipidaemia † 1  0/115 (0.00%)  1/115 (0.87%) 
Fluid retention † 1  0/115 (0.00%)  1/115 (0.87%) 
Hypercholesterolaemia † 1  2/115 (1.74%)  1/115 (0.87%) 
Hyperlipidaemia † 1  1/115 (0.87%)  1/115 (0.87%) 
Iron deficiency † 1  0/115 (0.00%)  1/115 (0.87%) 
Hyperglycaemia † 1  1/115 (0.87%)  0/115 (0.00%) 
Hyperkalaemia † 1  1/115 (0.87%)  0/115 (0.00%) 
Hyperuricaemia † 1  2/115 (1.74%)  0/115 (0.00%) 
Hypervitaminosis D † 1  1/115 (0.87%)  0/115 (0.00%) 
Hypoglycaemia † 1  1/115 (0.87%)  0/115 (0.00%) 
Hyponatraemia † 1  1/115 (0.87%)  0/115 (0.00%) 
Increased appetite † 1  1/115 (0.87%)  0/115 (0.00%) 
Metabolic acidosis † 1  1/115 (0.87%)  0/115 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain † 1  8/115 (6.96%)  11/115 (9.57%) 
Arthralgia † 1  5/115 (4.35%)  6/115 (5.22%) 
Foot deformity † 1  2/115 (1.74%)  3/115 (2.61%) 
Growing pains † 1  0/115 (0.00%)  1/115 (0.87%) 
Bone pain † 1  2/115 (1.74%)  0/115 (0.00%) 
Pain in extremity † 1  14/115 (12.17%)  10/115 (8.70%) 
Myalgia † 1  0/115 (0.00%)  3/115 (2.61%) 
Musculoskeletal chest pain † 1  2/115 (1.74%)  2/115 (1.74%) 
Musculoskeletal pain † 1  2/115 (1.74%)  2/115 (1.74%) 
Joint contracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Muscle atrophy † 1  0/115 (0.00%)  1/115 (0.87%) 
Muscle tightness † 1  0/115 (0.00%)  1/115 (0.87%) 
Muscular weakness † 1  1/115 (0.87%)  1/115 (0.87%) 
Osteoporosis † 1  0/115 (0.00%)  1/115 (0.87%) 
Scoliosis † 1  2/115 (1.74%)  1/115 (0.87%) 
Tendinous contracture † 1  0/115 (0.00%)  1/115 (0.87%) 
Joint crepitation † 1  1/115 (0.87%)  0/115 (0.00%) 
Lordosis † 1  1/115 (0.87%)  0/115 (0.00%) 
Muscle spasms † 1  1/115 (0.87%)  0/115 (0.00%) 
Osteopenia † 1  2/115 (1.74%)  0/115 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Skin papilloma † 1  2/115 (1.74%)  1/115 (0.87%) 
Nervous system disorders     
Headache † 1  21/115 (18.26%)  21/115 (18.26%) 
Autism † 1  0/115 (0.00%)  1/115 (0.87%) 
Coordination abnormal † 1  0/115 (0.00%)  1/115 (0.87%) 
Epilepsy † 1  0/115 (0.00%)  1/115 (0.87%) 
Migraine † 1  1/115 (0.87%)  1/115 (0.87%) 
Dizziness † 1  1/115 (0.87%)  0/115 (0.00%) 
Hypotonia † 1  1/115 (0.87%)  0/115 (0.00%) 
Sinus headache † 1  0/115 (0.00%)  2/115 (1.74%) 
Petit mal epilepsy † 1  0/115 (0.00%)  1/115 (0.87%) 
Psychomotor hyperactivity † 1  1/115 (0.87%)  1/115 (0.87%) 
Restless legs syndrome † 1  0/115 (0.00%)  1/115 (0.87%) 
Tremor † 1  0/115 (0.00%)  1/115 (0.87%) 
Psychiatric disorders     
Attention deficit/hyperactivity disorder † 1  1/115 (0.87%)  3/115 (2.61%) 
Aggression † 1  0/115 (0.00%)  2/115 (1.74%) 
Sleep disorder † 1  0/115 (0.00%)  2/115 (1.74%) 
Abnormal behaviour † 1  0/115 (0.00%)  1/115 (0.87%) 
Negativism † 1  0/115 (0.00%)  1/115 (0.87%) 
Obsessive-compulsive disorder † 1  1/115 (0.87%)  1/115 (0.87%) 
Anxiety † 1  2/115 (1.74%)  0/115 (0.00%) 
Dysphemia † 1  1/115 (0.87%)  0/115 (0.00%) 
Middle insomnia † 1  1/115 (0.87%)  0/115 (0.00%) 
Mood swings † 1  2/115 (1.74%)  0/115 (0.00%) 
Renal and urinary disorders     
Haematuria † 1  1/115 (0.87%)  7/115 (6.09%) 
Dysuria † 1  3/115 (2.61%)  2/115 (1.74%) 
Enuresis † 1  1/115 (0.87%)  2/115 (1.74%) 
Bladder trabeculation † 1  0/115 (0.00%)  1/115 (0.87%) 
Flank pain † 1  0/115 (0.00%)  1/115 (0.87%) 
Hypercalciuria † 1  1/115 (0.87%)  0/115 (0.00%) 
Myoglobinuria † 1  1/115 (0.87%)  0/115 (0.00%) 
Nephrolithiasis † 1  2/115 (1.74%)  0/115 (0.00%) 
Urinary incontinence † 1  1/115 (0.87%)  2/115 (1.74%) 
Polyuria † 1  0/115 (0.00%)  1/115 (0.87%) 
Renal cyst † 1  0/115 (0.00%)  1/115 (0.87%) 
Urine abnormality † 1  2/115 (1.74%)  1/115 (0.87%) 
Pollakiuria † 1  1/115 (0.87%)  0/115 (0.00%) 
Reproductive system and breast disorders     
Genital discomfort † 1  0/115 (0.00%)  1/115 (0.87%) 
Respiratory, thoracic and mediastinal disorders     
Cough † 1  13/115 (11.30%)  19/115 (16.52%) 
Epistaxis † 1  4/115 (3.48%)  7/115 (6.09%) 
Oropharyngeal pain † 1  6/115 (5.22%)  7/115 (6.09%) 
Nasal congestion † 1  2/115 (1.74%)  3/115 (2.61%) 
Rhinorrhoea † 1  3/115 (2.61%)  3/115 (2.61%) 
Wheezing † 1  0/115 (0.00%)  3/115 (2.61%) 
Asthmatic crisis † 1  0/115 (0.00%)  1/115 (0.87%) 
Productive cough † 1  0/115 (0.00%)  1/115 (0.87%) 
Sneezing † 1  0/115 (0.00%)  1/115 (0.87%) 
Dyspnoea † 1  1/115 (0.87%)  0/115 (0.00%) 
Sinus congestion † 1  2/115 (1.74%)  0/115 (0.00%) 
Sleep apnoea syndrome † 1  2/115 (1.74%)  0/115 (0.00%) 
Throat irritation † 1  1/115 (0.87%)  0/115 (0.00%) 
Skin and subcutaneous tissue disorders     
Blister † 1  0/115 (0.00%)  1/115 (0.87%) 
Acne † 1  1/115 (0.87%)  0/115 (0.00%) 
Dermatitis allergic † 1  1/115 (0.87%)  0/115 (0.00%) 
Rash † 1  4/115 (3.48%)  4/115 (3.48%) 
Ingrowing nail † 1  0/115 (0.00%)  2/115 (1.74%) 
Pruritus † 1  2/115 (1.74%)  2/115 (1.74%) 
Rash erythematous † 1  1/115 (0.87%)  2/115 (1.74%) 
Dry skin † 1  0/115 (0.00%)  1/115 (0.87%) 
Ecchymosis † 1  0/115 (0.00%)  1/115 (0.87%) 
Hair texture abnormal † 1  0/115 (0.00%)  1/115 (0.87%) 
Hirsutism † 1  0/115 (0.00%)  1/115 (0.87%) 
Seborrhoeic dermatitis † 1  0/115 (0.00%)  1/115 (0.87%) 
Skin burning sensation † 1  0/115 (0.00%)  1/115 (0.87%) 
Skin lesion † 1  0/115 (0.00%)  1/115 (0.87%) 
Skin mass † 1  0/115 (0.00%)  1/115 (0.87%) 
Erythema † 1  1/115 (0.87%)  0/115 (0.00%) 
Skin exfoliation † 1  1/115 (0.87%)  0/115 (0.00%) 
Skin hyperpigmentation † 1  1/115 (0.87%)  0/115 (0.00%) 
Vascular disorders     
Hypertension † 1  2/115 (1.74%)  1/115 (0.87%) 
Hypotension † 1  0/115 (0.00%)  1/115 (0.87%) 
Aortic dilatation † 1  1/115 (0.87%)  0/115 (0.00%) 
Flushing † 1  3/115 (2.61%)  0/115 (0.00%) 
Haematoma † 1  1/115 (0.87%)  0/115 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA v15.1
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Patient Advocacy
Organization: PTC Therapeutics, Inc.
Phone: 1-866-562-4620
EMail: medinfo@ptcbio.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT01826487    
Other Study ID Numbers: PTC124-GD-020-DMD
2012-004527-20 ( EudraCT Number )
First Submitted: March 26, 2013
First Posted: April 8, 2013
Results First Submitted: July 17, 2020
Results First Posted: August 4, 2020
Last Update Posted: August 4, 2020