Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 7 of 1063 for:    BMD

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

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: NCT00847379
Recruitment Status : Terminated
First Posted : February 19, 2009
Results First Posted : July 15, 2020
Last Update Posted : July 15, 2020
Sponsor:
Collaborator:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
PTC Therapeutics

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Intervention Drug: Ataluren
Enrollment 173
Recruitment Details A total of 173 participants were screened for eligibility to enter this extension study after completing the 48-week double-blind study PTC124-GD-007-DMD (NCT00592553). All participants met entry criteria and enrolled in this study.
Pre-assignment Details Participants were categorized by the treatment group to which they had been randomly assigned in Study PTC124-GD-007-DMD (NCT00592553).
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren
Hide Arm/Group Description Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit (Week 48) in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Period Title: Overall Study
Started 59 57 57
As-treated Population [1] 59 57 57
Completed 0 0 0
Not Completed 59 57 57
Reason Not Completed
Study terminated by Sponsor             59             57             57
[1]
All participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Total
Hide Arm/Group Description Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Total of all reporting groups
Overall Number of Baseline Participants 59 57 57 173
Hide Baseline Analysis Population Description
As-treated population included all participants who completed Study 007 and received greater than or equal to (≥) 1 dose of ataluren in Study 007e.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 59 participants 57 participants 57 participants 173 participants
9.4  (2.53) 9.7  (2.89) 9.3  (2.28) 9.5  (2.57)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 57 participants 57 participants 173 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
59
 100.0%
57
 100.0%
57
 100.0%
173
 100.0%
1.Primary Outcome
Title Number 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. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Time Frame Baseline (Week 48 of Study 007) up to Week 102
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 59 57 57 173
Measure Type: Count of Participants
Unit of Measure: Participants
Any AEs
52
  88.1%
49
  86.0%
48
  84.2%
149
  86.1%
Drug-related AEs
10
  16.9%
10
  17.5%
22
  38.6%
42
  24.3%
Serious AEs
0
   0.0%
1
   1.8%
2
   3.5%
3
   1.7%
Mild AEs
34
  57.6%
23
  40.4%
26
  45.6%
83
  48.0%
Moderate AEs
13
  22.0%
22
  38.6%
19
  33.3%
54
  31.2%
Severe AEs
5
   8.5%
4
   7.0%
3
   5.3%
12
   6.9%
2.Primary Outcome
Title Number of Participants With Clinically Significant Abnormal Laboratory Parameters
Hide Description Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
Time Frame Baseline (Week 48 of Study 007) up to Week 102
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 59 57 57 173
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
3.Secondary Outcome
Title Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60
Hide Description The 6MWD test was performed in a 30 meters long flat corridor, where the participant was 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 Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: meters
Baseline Number Analyzed 53 participants 50 participants 49 participants 152 participants
351.60  (130.312) 375.95  (98.976) 356.25  (109.369) 361.11  (113.760)
Change at Week 60 Number Analyzed 46 participants 46 participants 47 participants 139 participants
-17.23  (39.608) -10.50  (41.390) -7.06  (50.121) -11.56  (43.881)
4.Secondary Outcome
Title Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM)
Hide Description The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean activity period/day/visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that greater than (>) 2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was less than (<) 50 percent (%) of the mean active period across all days for that participant's visit.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: meters
Baseline Number Analyzed 52 participants 49 participants 48 participants 149 participants
759.715  (77.5210) 742.223  (107.7264) 747.464  (102.6531) 750.016  (96.1057)
Change at Week 60 Number Analyzed 41 participants 38 participants 40 participants 119 participants
1.946  (61.2247) 34.283  (88.1587) -1.866  (93.0531) 10.991  (82.6356)
5.Secondary Outcome
Title Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM
Hide Description The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/day/visit during the active periods was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: steps
Baseline Number Analyzed 52 participants 49 participants 48 participants 149 participants
4934.575  (2433.230) 4543.766  (2051.300) 5175.330  (2307.436) 4883.613  (2272.108)
Change at Week 60 Number Analyzed 41 participants 38 participants 40 participants 119 participants
-357.679  (1232.109) -215.062  (1761.773) -647.504  (1680.522) -409.558  (1566.386)
6.Secondary Outcome
Title Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM
Hide Description The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/hour during the active periods for the days in a visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: steps
Baseline Number Analyzed 52 participants 49 participants 48 participants 149 participants
395.491  (200.7671) 368.672  (155.6648) 420.684  (188.9124) 394.787  (183.0994)
Change at Week 60 Number Analyzed 41 participants 38 participants 40 participants 119 participants
-27.650  (87.8797) -34.693  (128.0609) -54.755  (122.4142) -39.010  (113.3481)
7.Secondary Outcome
Title Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM
Hide Description SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). The maximum continuous 10-minute, 20-minute, 30-minute, and 60-minute total step counts were computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: steps
Baseline: 10-minute total step count Number Analyzed 52 participants 49 participants 48 participants 149 participants
34.603  (12.4886) 32.863  (8.8533) 35.256  (10.0379) 34.241  (10.5911)
Change at Week 60: 10-minute total step count Number Analyzed 41 participants 38 participants 40 participants 119 participants
-1.140  (6.9584) -0.607  (7.2946) -2.372  (7.1735) -1.384  (7.1170)
Baseline: 20-minute total step count Number Analyzed 52 participants 49 participants 48 participants 149 participants
28.138  (11.0629) 26.096  (7.9168) 28.323  (9.3133) 27.526  (9.5426)
Change at Week 60: 20-minute total step count Number Analyzed 41 participants 38 participants 40 participants 119 participants
-1.459  (6.7165) -0.662  (6.2171) -2.510  (6.4346) -1.558  (6.4549)
Baseline: 30-minute total step count Number Analyzed 52 participants 49 participants 48 participants 149 participants
24.222  (9.9576) 22.205  (7.1045) 24.498  (8.4938) 23.648  (8.6306)
Change at Week 60: 30-minute total step count Number Analyzed 41 participants 38 participants 40 participants 119 participants
-1.400  (5.7605) -0.450  (5.6811) -2.457  (5.9579) -1.452  (5.8110)
Baseline: 60-minute total step count Number Analyzed 52 participants 49 participants 48 participants 149 participants
18.019  (7.8145) 16.453  (5.6625) 18.599  (6.9509) 17.691  (6.8991)
Change at Week 60: 60-minute total step count Number Analyzed 41 participants 38 participants 40 participants 119 participants
-0.977  (3.7328) -0.210  (4.7035) -2.257  (5.1038) -1.162  (4.5803)
8.Secondary Outcome
Title Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60
Hide Description SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear SAM for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again. Percentage of time during active periods spent at no activity (0 steps/min), low activity (≤15 steps/min), medium activity (16-30 steps/min), and high activity (>30 steps/min) were computed for each participant. For each day, an active period was defined as first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: percentage of time
Baseline: Time spent at no activity Number Analyzed 52 participants 49 participants 48 participants 149 participants
52.818  (14.4129) 53.372  (12.2352) 50.006  (12.5263) 52.094  (13.1188)
Change at Week 60: Time spent at no activity Number Analyzed 41 participants 38 participants 40 participants 119 participants
-0.051  (8.1776) 0.830  (11.2481) 2.194  (7.8652) 0.985  (9.1467)
Baseline: Time spent at low activity Number Analyzed 52 participants 49 participants 48 participants 149 participants
30.677  (7.8531) 31.170  (7.2431) 32.393  (7.2426) 31.392  (7.4462)
Change at Week 60: Time spent at low activity Number Analyzed 41 participants 38 participants 40 participants 119 participants
1.270  (5.8178) 1.172  (8.3050) 0.252  (5.5885) 0.896  (6.6058)
Baseline: Time spent at medium activity Number Analyzed 52 participants 49 participants 48 participants 149 participants
10.248  (4.7620) 10.119  (4.4344) 10.983  (4.6049) 10.442  (4.5901)
Change at Week 60: Time spent at medium activity Number Analyzed 41 participants 38 participants 40 participants 119 participants
-0.542  (2.0960) -1.356  (3.7830) -0.965  (3.3763) -0.944  (3.1392)
Baseline: Time spent at high activity Number Analyzed 51 participants 49 participants 48 participants 148 participants
6.404  (4.5938) 5.370  (3.4243) 6.673  (4.5847) 6.149  (4.2476)
Change at Week 60: Time spent at high activity Number Analyzed 39 participants 38 participants 40 participants 117 participants
-0.643  (2.2656) -0.520  (2.7196) -1.506  (2.9904) -0.898  (2.6913)
9.Secondary Outcome
Title Change From Baseline in Time to Stand From Supine Position at Week 60
Hide Description If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: seconds
Baseline Number Analyzed 53 participants 50 participants 49 participants 152 participants
13.736  (11.3353) 11.796  (10.3544) 12.398  (11.6010) 12.666  (11.0678)
Change at Week 60 Number Analyzed 49 participants 47 participants 48 participants 144 participants
0.665  (3.0080) -0.187  (4.6289) 0.002  (4.6322) 0.166  (4.1373)
10.Secondary Outcome
Title Change From Baseline in Time to Walk/Run 10 Meters at Week 60
Hide Description If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: seconds
Baseline Number Analyzed 53 participants 50 participants 49 participants 152 participants
8.143  (5.1475) 6.986  (2.9995) 6.967  (3.5789) 7.384  (4.0530)
Change at Week 60 Number Analyzed 49 participants 47 participants 48 participants 144 participants
1.441  (4.4822) 0.547  (2.2058) 0.758  (2.6862) 0.922  (3.2905)
11.Secondary Outcome
Title Change From Baseline in Time to Climb 4 Stairs at Week 60
Hide Description If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: seconds
Baseline Number Analyzed 53 participants 50 participants 49 participants 152 participants
9.077  (9.2543) 6.738  (6.4069) 8.071  (9.2057) 7.984  (8.4076)
Change at Week 60 Number Analyzed 49 participants 47 participants 48 participants 144 participants
0.831  (2.4664) 0.696  (4.0707) 0.194  (2.5268) 0.574  (3.0898)
12.Secondary Outcome
Title Change From Baseline in Time to Descend 4 Stairs at Week 60
Hide Description If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: seconds
Baseline Number Analyzed 53 participants 50 participants 49 participants 152 participants
7.685  (8.9934) 5.638  (5.5635) 6.641  (7.8591) 6.675  (7.6319)
Change at Week 60 Number Analyzed 49 participants 47 participants 48 participants 144 participants
1.167  (4.7624) 0.626  (5.2567) -0.648  (4.9202) 0.385  (5.0045)
13.Secondary Outcome
Title Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400
Hide Description The heart rate was measured with a Polar RS400 heart rate monitor, which consists of a transmitter strap worn around the chest and a wristwatch receiver. The monitor produces a digital text file with 1 value per minute that represents the mean heart rate for that minute. Mean heart rates values were collected prior to, during, and after the 6MWT. The participant rested for 5 minutes in a sitting position prior to the 6MWT, and the mean heart rate for the last minute of this rest period was collected and documented as the resting heart rate. During the 6MWT, the mean heart rate was collected and documented as the active heart rate. After completing the 6MWT and resting for 3 minutes, the mean heart rate for 1 minute was collected and documented as the recovery heart rate.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 53 50 49 152
Mean (Standard Deviation)
Unit of Measure: beats/minute
Baseline: Resting heart rate Number Analyzed 52 participants 50 participants 48 participants 150 participants
105.3  (13.29) 108.8  (10.71) 103.7  (12.98) 105.9  (12.48)
Change at Week 60: Resting heart rate Number Analyzed 45 participants 46 participants 43 participants 134 participants
4.0  (13.91) 3.7  (10.11) 4.8  (10.82) 4.2  (11.65)
Baseline: Active heart rate Number Analyzed 52 participants 48 participants 48 participants 148 participants
138.7  (21.45) 144.8  (14.55) 138.6  (20.87) 140.6  (19.36)
Change at Week 60: Active heart rate Number Analyzed 45 participants 44 participants 42 participants 131 participants
1.6  (17.65) 6.1  (15.45) 2.4  (14.87) 3.4  (16.07)
Baseline: Recovery heart rate Number Analyzed 52 participants 48 participants 48 participants 148 participants
110.0  (13.32) 112.0  (12.17) 109.4  (13.21) 110.4  (12.88)
Change at Week 60: Recovery heart rate Number Analyzed 43 participants 44 participants 44 participants 131 participants
2.1  (12.82) 4.6  (12.57) 2.3  (11.29) 3.0  (12.20)
14.Secondary Outcome
Title Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60
Hide Description Basic attention and working memory was measured using the digit span task. A series of digits (0-9) were presented to the child in an auditory format only. The task had 2 parts; in the forward condition, the child was requested to repeat back the digits in the order they were presented and in the backward condition, he was requested to reverse the order of presentation. A raw score of the total number of correct responses was converted to an age-scaled-score (z-score) by subtracting the corresponding mean and dividing by the corresponding standard deviation of a reference population for that age.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: z-score
Baseline: Forward condition Number Analyzed 56 participants 54 participants 51 participants 161 participants
4.1  (2.97) 3.6  (2.54) 3.2  (2.21) 3.6  (2.61)
Change at Week 60: Forward condition Number Analyzed 52 participants 51 participants 51 participants 154 participants
0.0  (1.53) 0.0  (1.19) 0.0  (1.22) 0.0  (1.32)
Baseline: Backward condition Number Analyzed 56 participants 54 participants 51 participants 161 participants
1.4  (0.98) 1.3  (0.82) 1.2  (0.66) 1.3  (0.84)
Change at Week 60: Backward condition Number Analyzed 49 participants 51 participants 49 participants 149 participants
0.1  (0.86) -0.1  (0.66) 0.0  (0.58) 0.0  (0.71)
15.Secondary Outcome
Title Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60
Hide Description HRQL was measured via the PedsQL. The generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and the fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Examples of items in each of the generic core module scales include: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline: Physical functioning score Number Analyzed 54 participants 54 participants 49 participants 157 participants
63.8145  (24.46918) 63.3019  (22.72039) 60.4865  (22.33484) 62.5995  (23.11654)
Change at Week 60: Physical functioning score Number Analyzed 50 participants 47 participants 49 participants 146 participants
-0.5982  (14.91137) -1.2538  (14.60384) 1.2755  (19.01169) -0.1804  (16.23570)
Baseline: Emotional functioning score Number Analyzed 54 participants 54 participants 49 participants 157 participants
76.852  (21.1530) 73.241  (20.1681) 73.265  (22.2568) 74.490  (21.1086)
Change at Week 60: Emotional functioning score Number Analyzed 50 participants 47 participants 49 participants 146 participants
-1.675  (13.6361) 2.128  (14.9181) -0.995  (13.1621) -0.223  (13.9106)
Baseline: Social functioning score Number Analyzed 54 participants 54 participants 50 participants 158 participants
72.5000  (21.60429) 68.6111  (21.37748) 71.3000  (21.32858) 70.7911  (21.36670)
Change at Week 60: Social functioning score Number Analyzed 50 participants 47 participants 50 participants 147 participants
-2.2000  (13.48317) 0.8511  (17.36185) 0.3500  (15.15237) -0.3571  (15.32468)
Baseline: School functioning score Number Analyzed 52 participants 54 participants 48 participants 154 participants
72.019  (20.7756) 71.111  (18.2918) 69.063  (20.3861) 70.779  (19.7213)
Change at Week 60: School functioning score Number Analyzed 47 participants 46 participants 47 participants 140 participants
-1.064  (16.5153) -1.630  (17.7029) 1.809  (16.9214) -0.286  (16.9931)
16.Secondary Outcome
Title Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60
Hide Description HRQL was measured via the PedsQL. The generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and the fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Examples of items in each of the generic core module scales include: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline: Physical functioning score Number Analyzed 57 participants 55 participants 56 participants 168 participants
56.8217  (23.80718) 52.5162  (20.02845) 52.6307  (24.35727) 54.0152  (22.78661)
Change at Week 60: Physical functioning score Number Analyzed 52 participants 49 participants 54 participants 155 participants
-0.8499  (12.75478) -1.1662  (13.27447) -0.1736  (14.75049) -0.7143  (13.55924)
Baseline: Emotional functioning score Number Analyzed 57 participants 55 participants 56 participants 168 participants
74.7368  (17.73870) 72.5455  (16.04025) 68.2813  (22.05309) 71.8676  (18.87211)
Change at Week 60: Emotional functioning score Number Analyzed 52 participants 49 participants 54 participants 155 participants
-1.9231  (11.11289) -0.3061  (13.32323) -1.9213  (16.86642) -1.4113  (13.95342)
Baseline: Social functioning score Number Analyzed 57 participants 55 participants 56 participants 168 participants
61.9518  (19.07606) 61.6136  (15.44605) 59.7545  (21.34593) 61.1086  (18.71300)
Change at Week 60: Social functioning score Number Analyzed 52 participants 49 participants 54 participants 155 participants
1.5144  (13.03356) -0.7908  (11.34994) 0.3472  (12.76238) 0.3790  (12.38287)
Baseline: School functioning score Number Analyzed 56 participants 55 participants 55 participants 166 participants
68.6607  (16.05363) 64.0000  (15.07389) 65.9091  (18.20894) 66.2048  (16.50694)
Change at Week 60: School functioning score Number Analyzed 50 participants 48 participants 53 participants 151 participants
0.8000  (9.60230) 1.6667  (10.93177) -1.0377  (12.60946) 0.4305  (11.13464)
17.Secondary Outcome
Title Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60
Hide Description HRQL was measured via the PedsQL. The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. PedsQL was completed by both the participant and/or a parent/caregiver. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time;" Each of the fatigue-specific module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 55 participants 53 participants 51 participants 159 participants
76.7968  (18.31310) 72.7833  (20.95237) 72.5947  (17.32956) 74.1111  (18.92041)
Change at Week 60 Number Analyzed 50 participants 46 participants 49 participants 145 participants
-2.0484  (9.77928) 0.3650  (15.94374) -1.8049  (11.12724) -1.2005  (12.42615)
18.Secondary Outcome
Title Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60
Hide Description HRQL was measured via the PedsQL. The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time;" Each of the fatigue-specific module items was scored on a 5-point likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 56 participants 53 participants 56 participants 165 participants
75.7864  (13.88014) 71.6596  (12.78488) 71.1062  (13.62739) 72.8724  (13.53355)
Change at Week 60 Number Analyzed 53 participants 46 participants 54 participants 153 participants
-0.3272  (6.64218) 1.2219  (10.11693) 0.2269  (8.55130) 0.3341  (8.44319)
19.Secondary Outcome
Title Change From Baseline in Participant and Parent/Caregiver Reported Activities of Daily Living of Participants Who Were Unable to Complete the 6MWT (Nonambulatory Participants), as Measured by the Egen Klassifikation (EK) Scale at Week 60
Hide Description Activities of daily living were measured using the EK scale in all participants who were unable to complete the 6MWT at Screening/Baseline on Day 1. 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 the arms, 6) ability to use the 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 performed the tasks of daily life (as described by Categories 1 to 9) and how he perceived his well-being (as described by Category 10).
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Non-Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and who had lost all independent ambulation prior to entering Study 007e. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 5 4 6 15
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Number Analyzed 4 participants 4 participants 5 participants 13 participants
3.0  (2.00) 4.3  (0.50) 3.2  (1.92) 3.5  (1.61)
Change at Week 60 Number Analyzed 4 participants 4 participants 5 participants 13 participants
1.5  (0.58) 0.8  (1.26) -0.8  (1.48) 0.4  (1.50)
20.Secondary Outcome
Title Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60
Hide Description TSQM consisted of 14 questions about treatment satisfaction with drug in 4 domains: Effectiveness (Questions 1-3 scored as 1 [extremely dissatisfied] to 7 [extremely satisfied]), Side Effects (question 4 scored as 0 [no] or 1 [yes]; question 5 scored as 1 [extremely bothersome] to 5 [not at all bothersome]; questions 6 - 8 scored as 1 [a great deal] to 5 [not at all]), Convenience (questions 9 and 10 scored as 1 [extremely difficult] to 7 [extremely easy]; question 11 scored as 1 [extremely inconvenient] to 5 [extremely convenient]) and Global Satisfaction (question 12 scored as 1 [not at all confident] to 7 [extremely confident]; question 13 scored as 1 [not at all certain] to 5 [extremely certain]; question 14 scored as 1 [extremely dissatisfied] to 5 [extremely satisfied]). The scores of each of the domains were added together and an algorithm was used to create a score of 0 to 100, with higher scores indicating better treatment satisfaction.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline: Effectiveness score Number Analyzed 53 participants 54 participants 52 participants 159 participants
56.8134  (26.72451) 53.8580  (21.53292) 50.4274  (22.96804) 53.7212  (23.82541)
Change at Week 60: Effectiveness score Number Analyzed 50 participants 46 participants 50 participants 146 participants
2.7778  (15.52191) 5.6159  (19.53233) 1.7778  (14.99307) 3.3295  (16.68460)
Baseline: Side-effects score Number Analyzed 55 participants 55 participants 54 participants 164 participants
96.8182  (10.81681) 97.7273  (7.64176) 96.8364  (8.94695) 97.1291  (9.18246)
Change at Week 60: Side-effects score Number Analyzed 51 participants 47 participants 48 participants 146 participants
0.7353  (12.16084) -1.0638  (7.74500) -1.2587  (10.84441) -0.4994  (10.43907)
Baseline: Convenience score Number Analyzed 57 participants 55 participants 54 participants 166 participants
57.4074  (16.10609) 57.9798  (19.05889) 60.5967  (16.65858) 58.6345  (17.26216)
Change at Week 60: Convenience score Number Analyzed 53 participants 47 participants 50 participants 150 participants
0.8386  (12.39372) -1.6548  (11.75175) 1.3333  (9.80472) 0.2222  (11.38059)
Baseline: Global satisfaction score Number Analyzed 55 participants 55 participants 54 participants 164 participants
61.4286  (25.95857) 60.4870  (23.30835) 56.7791  (21.25768) 59.5819  (23.54109)
Change at Week 60: Global satisfaction score Number Analyzed 53 participants 47 participants 50 participants 150 participants
-0.2695  (13.57882) 1.2538  (15.30213) -0.4643  (14.78188) 0.1429  (14.45922)
21.Secondary Outcome
Title Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 60
Hide Description Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome.
Time Frame Baseline (Week 48 of Study 007), Week 60
Hide Outcome Measure Data
Hide Analysis Population Description
Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 58 56 56 170
Mean (Standard Deviation)
Unit of Measure: units/liter (U/L)
Baseline Number Analyzed 57 participants 56 participants 54 participants 167 participants
9163.1  (5542.80) 10009.1  (5828.26) 9150.7  (5657.67) 9442.8  (5656.99)
Change at Week 60 Number Analyzed 49 participants 51 participants 53 participants 153 participants
-455.1  (4962.00) -520.5  (6164.96) -334.3  (4810.45) -435.1  (5310.37)
22.Secondary Outcome
Title Study Drug Compliance
Hide Description Study drug compliance was assessed by participant daily diary and 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 (Week 48 of Study 007) to Week 96
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 59 57 57 173
Median (Full Range)
Unit of Measure: percentage of drug
0.00
(0.0 to 45.1)
0.00
(0.0 to 10.5)
0.00
(0.0 to 36.1)
0.00
(0.0 to 45.1)
23.Secondary Outcome
Title Trough Ataluren Plasma Concentration
Hide Description Plasma samples for the determination of ataluren concentrations were analyzed at the bioanalytical laboratory for ataluren parent drug using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation (LLOQ) of 0.5 micrograms/millilitre (mcg/mL). Values below the LLOQ were set to 0.
Time Frame Pre-morning dose (0 hour) at Baseline (Week 48 of 007 study), Weeks 54, 60, 72, 84, and 96
Hide Outcome Measure Data
Hide Analysis Population Description
As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e. 'Number analyzed'=participants evaluable at specified timepoints.
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description:
Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Number of Participants Analyzed 59 57 57 173
Mean (Standard Deviation)
Unit of Measure: mcg/mL
Week 48 Number Analyzed 58 participants 57 participants 57 participants 172 participants
12.656  (10.8951) 4.541  (3.4381) 0.000  (0.0000) 5.773  (8.4333)
Week 54 Number Analyzed 58 participants 57 participants 56 participants 171 participants
10.801  (8.4858) 13.941  (14.5222) 11.564  (9.3312) 12.097  (11.1172)
Week 60 Number Analyzed 55 participants 54 participants 56 participants 165 participants
11.547  (9.6034) 13.830  (11.9096) 13.302  (9.4765) 12.890  (10.3574)
Week 72 Number Analyzed 25 participants 21 participants 19 participants 65 participants
12.279  (10.5894) 13.930  (10.6393) 10.948  (6.3465) 12.424  (9.4948)
Week 84 Number Analyzed 6 participants 7 participants 5 participants 18 participants
15.232  (8.8909) 14.773  (10.4404) 11.082  (14.6331) 13.901  (10.7415)
Week 96 Number Analyzed 1 participants 2 participants 0 participants 3 participants
8.430 5.685  (2.7648) 6.600  (2.5167)
Time Frame Baseline (Week 48 of Study 007) up to Week 102
Adverse Event Reporting Description As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
 
Arm/Group Title High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Hide Arm/Group Description Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated. All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
All-Cause Mortality
High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Hide Serious Adverse Events
High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/59 (0.00%)      1/57 (1.75%)      2/57 (3.51%)      3/173 (1.73%)    
Injury, poisoning and procedural complications         
Femur fracture  1  0/59 (0.00%)  1/57 (1.75%)  1/57 (1.75%)  2/173 (1.16%) 
Vascular disorders         
Hypertension  1  0/59 (0.00%)  0/57 (0.00%)  1/57 (1.75%)  1/173 (0.58%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
High-Dose Ataluren/High-Dose Ataluren Low-Dose Ataluren/High-Dose Ataluren Placebo/High-Dose Ataluren Overall Participants: High-Dose Ataluren
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   53/59 (89.83%)      51/57 (89.47%)      51/57 (89.47%)      155/173 (89.60%)    
Blood and lymphatic system disorders         
Lymphadenopathy  1  0/59 (0.00%)  0 1/57 (1.75%)  1 2/57 (3.51%)  2 3/173 (1.73%)  3
Anaemia  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Microcytic anaemia  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Neutropenia  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Cardiac disorders         
Dilatation ventricular  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  1 2/173 (1.16%)  2
Arrhythmia  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Cardiomyopathy  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Left ventricular hypertrophy  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Tachycardia  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Congenital, familial and genetic disorders         
Kidney malformation  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Ear and labyrinth disorders         
Deafness  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Ear pain  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Tympanic membrane hyperaemia  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Vertigo  1  1/59 (1.69%)  5 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  5
Endocrine disorders         
Cushingoid  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Eye disorders         
Cataract  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Myopia  1  1/59 (1.69%)  1 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  2
Abnormal sensation in eye  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Conjunctivitis  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Hypermetropia  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Photophobia  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Strabismus  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Vision blurred  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Gastrointestinal disorders         
Vomiting  1  11/59 (18.64%)  16 12/57 (21.05%)  23 20/57 (35.09%)  33 43/173 (24.86%)  72
Diarrhoea  1  3/59 (5.08%)  4 11/57 (19.30%)  13 6/57 (10.53%)  8 20/173 (11.56%)  25
Abdominal pain  1  6/59 (10.17%)  10 4/57 (7.02%)  4 6/57 (10.53%)  7 16/173 (9.25%)  21
Abdominal pain upper  1  2/59 (3.39%)  2 9/57 (15.79%)  11 4/57 (7.02%)  5 15/173 (8.67%)  18
Flatulence  1  7/59 (11.86%)  7 4/57 (7.02%)  4 3/57 (5.26%)  3 14/173 (8.09%)  14
Nausea  1  5/59 (8.47%)  5 3/57 (5.26%)  4 3/57 (5.26%)  3 11/173 (6.36%)  12
Dyspepsia  1  1/59 (1.69%)  1 2/57 (3.51%)  2 2/57 (3.51%)  2 5/173 (2.89%)  5
Constipation  1  1/59 (1.69%)  1 0/57 (0.00%)  0 3/57 (5.26%)  3 4/173 (2.31%)  4
Stomach discomfort  1  1/59 (1.69%)  1 2/57 (3.51%)  3 1/57 (1.75%)  1 4/173 (2.31%)  5
Aerophagia  1  1/59 (1.69%)  1 0/57 (0.00%)  0 2/57 (3.51%)  2 3/173 (1.73%)  3
Gastrooesophageal reflux disease  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Abdominal discomfort  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  2 1/173 (0.58%)  2
Abdominal pain lower  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Abdominal tenderness  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Abnormal faeces  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Aphthous stomatitis  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Chapped lips  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Duodenogastric reflux  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Eructation  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Faecal incontinence  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Gastritis  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Gastrointestinal sounds abnormal  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Gingivitis  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Oral pain  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Tongue disorder  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
General disorders         
Disease progression  1  9/59 (15.25%)  9 7/57 (12.28%)  7 3/57 (5.26%)  3 19/173 (10.98%)  19
Asthenia  1  2/59 (3.39%)  3 5/57 (8.77%)  5 2/57 (3.51%)  2 9/173 (5.20%)  10
Gait disturbance  1  4/59 (6.78%)  4 3/57 (5.26%)  3 2/57 (3.51%)  2 9/173 (5.20%)  9
Pyrexia  1  4/59 (6.78%)  4 2/57 (3.51%)  3 3/57 (5.26%)  4 9/173 (5.20%)  11
Fatigue  1  3/59 (5.08%)  3 1/57 (1.75%)  1 1/57 (1.75%)  1 5/173 (2.89%)  5
Malaise  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Oedema peripheral  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Immune system disorders         
Hypersensitivity  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  1 2/173 (1.16%)  2
Infections and infestations         
Influenza  1  7/59 (11.86%)  7 5/57 (8.77%)  5 6/57 (10.53%)  6 18/173 (10.40%)  18
Nasopharyngitis  1  3/59 (5.08%)  4 8/57 (14.04%)  10 5/57 (8.77%)  5 16/173 (9.25%)  19
Upper respiratory tract infection  1  6/59 (10.17%)  8 3/57 (5.26%)  4 5/57 (8.77%)  5 14/173 (8.09%)  17
Ear infection  1  2/59 (3.39%)  3 2/57 (3.51%)  2 4/57 (7.02%)  5 8/173 (4.62%)  10
Gastroenteritis viral  1  3/59 (5.08%)  3 1/57 (1.75%)  2 3/57 (5.26%)  3 7/173 (4.05%)  8
Gastroenteritis  1  2/59 (3.39%)  2 0/57 (0.00%)  0 3/57 (5.26%)  4 5/173 (2.89%)  6
Otitis media  1  2/59 (3.39%)  2 2/57 (3.51%)  3 1/57 (1.75%)  1 5/173 (2.89%)  6
Rhinitis  1  1/59 (1.69%)  1 1/57 (1.75%)  1 2/57 (3.51%)  2 4/173 (2.31%)  4
Bronchitis  1  0/59 (0.00%)  0 1/57 (1.75%)  1 2/57 (3.51%)  2 3/173 (1.73%)  3
Croup infectious  1  1/59 (1.69%)  1 0/57 (0.00%)  0 2/57 (3.51%)  2 3/173 (1.73%)  3
Lower respiratory tract infection  1  0/59 (0.00%)  0 0/57 (0.00%)  0 3/57 (5.26%)  4 3/173 (1.73%)  4
Paronychia  1  1/59 (1.69%)  1 1/57 (1.75%)  1 1/57 (1.75%)  1 3/173 (1.73%)  3
Sinusitis  1  0/59 (0.00%)  0 2/57 (3.51%)  2 1/57 (1.75%)  4 3/173 (1.73%)  6
Urinary tract infection  1  0/59 (0.00%)  0 1/57 (1.75%)  1 2/57 (3.51%)  2 3/173 (1.73%)  3
Viral infection  1  1/59 (1.69%)  1 0/57 (0.00%)  0 2/57 (3.51%)  2 3/173 (1.73%)  3
Enterobiasis  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  1 2/173 (1.16%)  2
Gastrointestinal infection  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  2 2/173 (1.16%)  3
Molluscum contagiosum  1  1/59 (1.69%)  1 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  2
Pharyngitis  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Pharyngitis streptococcal  1  2/59 (3.39%)  3 0/57 (0.00%)  0 0/57 (0.00%)  0 2/173 (1.16%)  3
Tinea pedis  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  3 2/173 (1.16%)  4
Conjunctivitis infective  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Helicobacter infection  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Localised infection  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Lung infection  1  0/59 (0.00%)  0 1/57 (1.75%)  2 0/57 (0.00%)  0 1/173 (0.58%)  2
Onychomycosis  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Oral herpes  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Otitis externa  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Pneumonia  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Scarlet fever  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Injury, poisoning and procedural complications         
Fall  1  5/59 (8.47%)  6 5/57 (8.77%)  5 4/57 (7.02%)  4 14/173 (8.09%)  15
Contusion  1  2/59 (3.39%)  3 1/57 (1.75%)  1 0/57 (0.00%)  0 3/173 (1.73%)  4
Iliotibial band syndrome  1  1/59 (1.69%)  1 2/57 (3.51%)  2 0/57 (0.00%)  0 3/173 (1.73%)  3
Joint sprain  1  1/59 (1.69%)  1 2/57 (3.51%)  2 0/57 (0.00%)  0 3/173 (1.73%)  3
Arthropod bite  1  2/59 (3.39%)  2 0/57 (0.00%)  0 0/57 (0.00%)  0 2/173 (1.16%)  2
Femur fracture  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Limb injury  1  1/59 (1.69%)  1 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  2
Lower limb fracture  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Arthropod sting  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Excoriation  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Facial bones fracture  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Foot fracture  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Head injury  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Humerus fracture  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Incision site erythema  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Lumbar vertebral fracture  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Mouth injury  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Muscle strain  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Overdose  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Procedural pain  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin laceration  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Spinal compression fracture  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Wound  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Wound dehiscence  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Investigations         
Blood sodium increased  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  1 2/173 (1.16%)  2
Weight decreased  1  0/59 (0.00%)  0 2/57 (3.51%)  2 0/57 (0.00%)  0 2/173 (1.16%)  2
Blood bicarbonate decreased  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Blood triglycerides increased  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Breath sounds abnormal  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Weight increased  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Metabolism and nutrition disorders         
Decreased appetite  1  2/59 (3.39%)  2 1/57 (1.75%)  1 1/57 (1.75%)  1 4/173 (2.31%)  4
Dehydration  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Hypovitaminosis  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Insulin resistance  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Iron deficiency  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Musculoskeletal and connective tissue disorders         
Back pain  1  4/59 (6.78%)  11 3/57 (5.26%)  3 4/57 (7.02%)  7 11/173 (6.36%)  21
Muscular weakness  1  5/59 (8.47%)  6 4/57 (7.02%)  4 2/57 (3.51%)  2 11/173 (6.36%)  12
Pain in extremity  1  5/59 (8.47%)  8 4/57 (7.02%)  4 1/57 (1.75%)  1 10/173 (5.78%)  14
Joint contracture  1  4/59 (6.78%)  4 4/57 (7.02%)  8 1/57 (1.75%)  1 9/173 (5.20%)  13
Arthralgia  1  3/59 (5.08%)  3 4/57 (7.02%)  4 0/57 (0.00%)  0 7/173 (4.05%)  7
Muscle tightness  1  2/59 (3.39%)  2 3/57 (5.26%)  3 1/57 (1.75%)  1 6/173 (3.47%)  6
Lordosis  1  2/59 (3.39%)  2 1/57 (1.75%)  1 2/57 (3.51%)  2 5/173 (2.89%)  5
Musculoskeletal chest pain  1  2/59 (3.39%)  2 1/57 (1.75%)  1 2/57 (3.51%)  2 5/173 (2.89%)  5
Myalgia  1  2/59 (3.39%)  3 2/57 (3.51%)  2 1/57 (1.75%)  1 5/173 (2.89%)  6
Muscle spasms  1  1/59 (1.69%)  1 3/57 (5.26%)  3 0/57 (0.00%)  0 4/173 (2.31%)  4
Osteoporosis  1  3/59 (5.08%)  3 0/57 (0.00%)  0 0/57 (0.00%)  0 3/173 (1.73%)  3
Tendinous contracture  1  1/59 (1.69%)  1 2/57 (3.51%)  2 0/57 (0.00%)  0 3/173 (1.73%)  3
Muscle atrophy  1  1/59 (1.69%)  1 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  2
Scoliosis  1  0/59 (0.00%)  0 2/57 (3.51%)  2 0/57 (0.00%)  0 2/173 (1.16%)  2
Tendon disorder  1  0/59 (0.00%)  0 1/57 (1.75%)  1 1/57 (1.75%)  1 2/173 (1.16%)  2
Coccydynia  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Groin pain  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Growth retardation  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Joint instability  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Muscle contracture  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Musculoskeletal pain  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Neck pain  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Musculoskeletal stiffness  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Skin papilloma  1  1/59 (1.69%)  1 4/57 (7.02%)  5 1/57 (1.75%)  1 6/173 (3.47%)  7
Melanocytic naevus  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Nervous system disorders         
Headache  1  5/59 (8.47%)  48 15/57 (26.32%)  48 14/57 (24.56%)  17 34/173 (19.65%)  113
Migraine  1  2/59 (3.39%)  2 1/57 (1.75%)  1 1/57 (1.75%)  1 4/173 (2.31%)  4
Areflexia  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Dizziness  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Hypertonia  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Hypotonia  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Memory impairment  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Partial seizures  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Sedation  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Sinus headache  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Psychiatric disorders         
Attention deficit/hyperactivity disorder  1  2/59 (3.39%)  2 2/57 (3.51%)  2 1/57 (1.75%)  1 5/173 (2.89%)  5
Abnormal behaviour  1  0/59 (0.00%)  0 3/57 (5.26%)  3 0/57 (0.00%)  0 3/173 (1.73%)  3
Aggression  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Obsessive-compulsive disorder  1  0/59 (0.00%)  0 0/57 (0.00%)  0 2/57 (3.51%)  2 2/173 (1.16%)  2
Tic  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  2 2/173 (1.16%)  3
Depression  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Frustration  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Oppositional defiant disorder  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Renal and urinary disorders         
Enuresis  1  2/59 (3.39%)  2 3/57 (5.26%)  3 1/57 (1.75%)  1 6/173 (3.47%)  6
Urine abnormality  1  1/59 (1.69%)  1 0/57 (0.00%)  0 2/57 (3.51%)  2 3/173 (1.73%)  3
Pollakiuria  1  1/59 (1.69%)  1 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  2
Urinary incontinence  1  2/59 (3.39%)  2 0/57 (0.00%)  0 0/57 (0.00%)  0 2/173 (1.16%)  2
Dysuria  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Haematuria  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Kidney enlargement  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Micturition urgency  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Pyelocaliectasis  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Renal cyst  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Ureteric dilatation  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Respiratory, thoracic and mediastinal disorders         
Cough  1  6/59 (10.17%)  6 1/57 (1.75%)  1 6/57 (10.53%)  6 13/173 (7.51%)  13
Oropharyngeal pain  1  4/59 (6.78%)  6 2/57 (3.51%)  2 1/57 (1.75%)  1 7/173 (4.05%)  9
Nasal congestion  1  2/59 (3.39%)  2 2/57 (3.51%)  2 2/57 (3.51%)  2 6/173 (3.47%)  6
Epistaxis  1  3/59 (5.08%)  3 1/57 (1.75%)  2 0/57 (0.00%)  0 4/173 (2.31%)  5
Wheezing  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Asthma  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Dyspnoea  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Pharyngeal erythema  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Postnasal drip  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Productive cough  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Respiratory tract congestion  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Rhinitis allergic  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Sinus congestion  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Snoring  1  0/59 (0.00%)  0 0/57 (0.00%)  0 1/57 (1.75%)  1 1/173 (0.58%)  1
Skin and subcutaneous tissue disorders         
Rash  1  2/59 (3.39%)  2 1/57 (1.75%)  1 3/57 (5.26%)  3 6/173 (3.47%)  6
Dry skin  1  0/59 (0.00%)  0 1/57 (1.75%)  1 2/57 (3.51%)  2 3/173 (1.73%)  3
Ecchymosis  1  2/59 (3.39%)  2 0/57 (0.00%)  0 0/57 (0.00%)  0 2/173 (1.16%)  2
Eczema  1  1/59 (1.69%)  2 1/57 (1.75%)  1 0/57 (0.00%)  0 2/173 (1.16%)  3
Pruritus  1  1/59 (1.69%)  1 0/57 (0.00%)  0 1/57 (1.75%)  1 2/173 (1.16%)  2
Acne  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Cold sweat  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Hyperkeratosis  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Keratosis pilaris  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Petechiae  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Pityriasis rosea  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Rash maculo-papular  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Rash papular  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin chapped  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin depigmentation  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin discolouration  1  0/59 (0.00%)  0 1/57 (1.75%)  1 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin exfoliation  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Skin odour abnormal  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Swelling face  1  1/59 (1.69%)  1 0/57 (0.00%)  0 0/57 (0.00%)  0 1/173 (0.58%)  1
Vascular disorders         
Hot flush  1  0/59 (0.00%)  0 1/57 (1.75%)  2 0/57 (0.00%)  0 1/173 (0.58%)  2
Hypertension  1  0/59 (0.00%)  0 2/57 (3.51%)  2 2/57 (3.51%)  2 4/173 (2.31%)  4
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 11.0
This study was prematurely terminated by Sponsor per Data Monitoring Committee (DMC) recommendation to discontinue ongoing studies of high-dose ataluren in nmDBMD due to lack of efficacy for the high-dose ataluren.
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
Layout table for additonal information
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT00847379    
Other Study ID Numbers: PTC124-GD-007e-DMD
First Submitted: February 16, 2009
First Posted: February 19, 2009
Results First Submitted: June 26, 2020
Results First Posted: July 15, 2020
Last Update Posted: July 15, 2020