Trial Outcomes & Findings for Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) (NCT NCT00759876)

Last Updated: 2020-10-29

Results Overview

TEAE: any untoward medical occurrence or undesirable event(s) that begins or worsens following administration of the study drug, whether or not considered related to the treatment by the Investigator. Severity of an adverse event (AE) was classified as: mild (does not interfere with usual function), moderate (interferes with usual function; may require medical intervention), severe (interferes significantly with usual function; likely require medical intervention), life-threatening, and fatal. 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. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

36 participants

Primary outcome timeframe

Baseline up to Week 89

Results posted on

2020-10-29

Participant Flow

This extension study was initiated approximately 2 years after the completion of Study PTC124-GD-004-DMD (Study 004 \[NCT00264888\]).

Of the 36 participants, 11 received 20-, 20-, and 40-milligrams/kilograms (mg/kg); 20 received 10-, 10-, and 20-mg/kg; and 5 received 4-, 4-, and 8-mg/kg ataluren dose level in Study 004. The 31 participants receiving corticosteroid treatment at study entry could continue corticosteroid treatment during the study as long as regimen remained stable.

Participant milestones

Participant milestones
Measure	Ataluren Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Overall Study STARTED	36
Overall Study As-Treated Population	36
Overall Study Evaluable Population	35
Overall Study Ambulatory Evaluable Population	24
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	36

Reasons for withdrawal

Reasons for withdrawal
Measure	Ataluren Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Overall Study Sponsor terminated study early	36

Baseline Characteristics

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Age, Continuous	11.6 years STANDARD_DEVIATION 2.61 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline up to Week 89

Population: All enrolled participants who received at least 1 dose of study drug (As-Treated Population).

Outcome measures

Outcome measures
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) At least 1 TEAE	36 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Mild TEAE	9 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Moderate TEAE	16 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Severe TEAE	10 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Life-Threatening TEAE	1 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Fatal TEAE	0 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Related TEAE	28 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Serious TEAE	6 Participants

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable 6MWT data. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

The 6MWD was assessed in participants who were ambulatory using standardized procedures. Participants were not permitted to use assistive devices during the 6MWD test. Only the results of the participant's best valid test at each visit were included in the analysis. The mean change from baseline in the distance the participant walked is reported.

Outcome measures

Outcome measures
Measure	Ataluren n=23 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) Baseline	367.8 meters Standard Deviation 107.3
Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) Change from Baseline at Week 48	-80.4 meters Standard Deviation 59.5
Change From Baseline in 6-Minute Walk Distance (6MWD) as Measured by the 6-minute Walk Test (6MWT) Change from Baseline at Week 60	-101.5 meters Standard Deviation 64.4

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable data for the applicable timed function test. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Timed function tests included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. Timed function tests were assessed in ambulatory participants. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. Test results were set to missing in the analysis for participants who could not perform the tests for reasons other than PD (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Descend 4 Stairs, Change from Baseline at Week 48	5.25 seconds Standard Deviation 8.839
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Rise to Standing, Baseline	13.57 seconds Standard Deviation 11.111
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Rise to Standing, Change from Baseline at Week 48	6.49 seconds Standard Deviation 8.059
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Rise to Standing, Change from Baseline at Week 60	5.77 seconds Standard Deviation 7.790
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Walk/Run 10 m, Baseline	7.62 seconds Standard Deviation 3.641
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Walk/Run 10 m, Change from Baseline at Week 48	3.15 seconds Standard Deviation 5.591
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Walk/Run 10 m, Change from Baseline at Week 60	3.20 seconds Standard Deviation 5.930
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Ascend 4 Stairs, Baseline	7.16 seconds Standard Deviation 7.481
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Ascend 4 Stairs, Change from Baseline at Week 48	4.92 seconds Standard Deviation 6.856
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Ascend 4 Stairs, Change from Baseline at Week 60	6.30 seconds Standard Deviation 7.926
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Descend 4 Stairs, Baseline	6.42 seconds Standard Deviation 7.565
Change From Baseline in Proximal Muscle Function as Assessed by Speed During Timed Function Tests Descend 4 Stairs, Change from Baseline at Week 60	4.28 seconds Standard Deviation 7.275

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for standing from supine. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used for standing from supine position: 1) Unable to stand from supine, even with use of a chair. 2) Assisted Gowers, requires furniture to rise from supine to full upright posture. 3) Full Gowers, rolls over, stands with both hands "climbing up" legs to above knees to achieve full upright posture. 4) Half Gowers, rolls over, stands up with 1 hand support on lower legs. 5) Rolls to side and/or stands with 1 or both hands on floor to start to rise but does not touch legs. 6) Stands without rolling over or using hands. Increases from baseline are indicative of improving ability to perform functional task. If a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function Tests Baseline	3.25 scores on a scale Standard Deviation 1.260
Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 48	-0.58 scores on a scale Standard Deviation 0.929
Change From Baseline in Standing From Supine Position as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 60	-0.62 scores on a scale Standard Deviation 0.921

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for running/walking 10 meters. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to run/walk 10-meters: 1) Unable to walk independently. 2) Unable to walk independently but can walk with knee-ankle-foot orthoses (KAFOs) or with support from a person 3) Highly adapted, wide-based lordotic gait, cannot increase walking speed. 4) Moderately adapted gait, can pick up speed but cannot run. 5) Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground). 6) Runs and gets both feet off the ground (with no double-stance phase). At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function Tests Baseline	4.42 scores on a scale Standard Deviation 0.974
Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 48	-0.25 scores on a scale Standard Deviation 1.073
Change From Baseline in Run/Walk 10-Meters as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 60	-0.57 scores on a scale Standard Deviation 1.207

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for ascending 4 stairs. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to ascend 4 stairs: 1) Unable to climb 4 standard stairs. 2) Climbs 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Climbs 4 standard stairs "marking time" using 1 arm on 1 handrail. 4) Climbs 4 standard stairs "marking time" not needing handrail. 5) Climbs 4 standard stairs alternating feet, needs handrail for support. 6) Climbs 4 standard stairs alternating feet, not needing handrail support. At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function Tests Baseline	3.58 scores on a scale Standard Deviation 1.717
Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 48	-0.46 scores on a scale Standard Deviation 1.141
Change From Baseline in Ascending 4 Stairs as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 60	-0.48 scores on a scale Standard Deviation 1.365

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable method data for descending 4 stairs. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Timed test evaluations included scoring of method that ambulatory participants used to complete test. Scale for method used to descend 4 stairs: 1) Unable to descend 4 standard stairs. 2) Descends 4 standard stairs "marking time" (climbs 1 foot at a time, with both feet on a step before moving to next step), using both arms on 1 or both handrails. 3) Descends 4 standard stairs "marking time", using 1 arm on 1 handrail. 4) Descends 4 standard stairs "marking time", not needing handrail. 5) Descends 4 standard stairs alternating feet in both directions, needs handrail for support. 6) Descends 4 standard stairs alternating feet, not needing handrail support. At the visit, if a participant could not perform a timed function test because of PD, a method value of "1" was assigned in analysis. Test results set to missing in analysis for participants who could not perform tests for reasons other than PD (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function Tests Baseline	3.63 scores on a scale Standard Deviation 1.740
Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 48	-0.04 scores on a scale Standard Deviation 1.160
Change From Baseline in Descending 4 Stairs as Assessed by Method Scores During Timed Function Tests Change from Baseline at Week 60	-0.10 scores on a scale Standard Deviation 0.889

SECONDARY outcome

Timeframe: Baseline, Week (Wk) 48 and Wk 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable myometry data.

Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. With this system, evaluators judged the strength of each muscle using an 11-point descriptive scoring system. From the individual muscle-group scores, a total composite score was derived. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. The best of the 3 replicates was used in the analysis. An increase from Baseline is reflective of increased muscle strength, whereas a decrease from Baseline is reflective of decreased muscle strength. Participants who became unable to perform a myometry test because of disease progression were assigned a value of 0 for each visit at which the participant was no longer able to perform the test. Test results were set to missing in the analysis for participants who could not perform the tests for reasons other than disease progression (for example, bone fracture).

Outcome measures

Outcome measures
Measure	Ataluren n=35 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Knee Extension, Change from Baseline at Wk 60	-1.00 pounds Standard Deviation 1.567
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry Left (L) Knee Flexion, Baseline	9.48 pounds Standard Deviation 4.395
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Knee Flexion, Change from Baseline at Wk 48	1.23 pounds Standard Deviation 2.715
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Knee Flexion, Change from Baseline at Wk 60	0.64 pounds Standard Deviation 3.079
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry Right (R) Knee Flexion, Baseline	10.15 pounds Standard Deviation 5.439
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Knee Flexion, Change from Baseline at Wk 48	0.81 pounds Standard Deviation 2.254
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Knee Flexion, Change from Baseline at Wk 60	-0.05 pounds Standard Deviation 2.240
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Knee Extension, Baseline	8.63 pounds Standard Deviation 5.124
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Knee Extension, Change from Baseline at Wk 48	-1.07 pounds Standard Deviation 1.842
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Knee Extension, Baseline	9.10 pounds Standard Deviation 6.109
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Knee Extension, Change from Baseline at Wk 48	-1.33 pounds Standard Deviation 2.021
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Knee Extension, Change from Baseline at Wk 60	-1.65 pounds Standard Deviation 2.204
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Flexion, Baseline	5.70 pounds Standard Deviation 3.611
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Flexion, Change from Baseline at Wk 48	-0.15 pounds Standard Deviation 0.998
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Flexion, Change from Baseline at Wk 60	-0.51 pounds Standard Deviation 1.121
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Flexion, Baseline	5.85 pounds Standard Deviation 3.758
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Flexion, Change from Baseline at Wk 48	-0.28 pounds Standard Deviation 1.050
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Flexion, Change from Baseline at Wk 60	-0.55 pounds Standard Deviation 1.061
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Extension, Baseline	4.55 pounds Standard Deviation 2.322
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Extension, Change from Baseline at Wk 48	-0.10 pounds Standard Deviation 0.993
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Elbow Extension, Change from Baseline at Wk 60	-0.32 pounds Standard Deviation 0.818
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Extension, Baseline	4.89 pounds Standard Deviation 2.680
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Extension, Change from Baseline at Wk 48	-0.10 pounds Standard Deviation 1.600
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Elbow Extension, Change from Baseline at Wk 60	-0.60 pounds Standard Deviation 1.352
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Shoulder Abduction, Baseline	4.29 pounds Standard Deviation 2.783
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Shoulder Abduction Change from Baseline at Wk 48	-0.90 pounds Standard Deviation 0.906
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Shoulder Abduction Change from Baseline at Wk 60	-1.10 pounds Standard Deviation 1.632
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Shoulder Abduction, Baseline	4.39 pounds Standard Deviation 2.666
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Shoulder Abduction Change from Baseline at Wk 48	-1.02 pounds Standard Deviation 1.369
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Shoulder Abduction Change from Baseline at Wk 60	-1.23 pounds Standard Deviation 1.888
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Hand Grip, Baseline	15.19 pounds Standard Deviation 5.881
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Hand Grip, Change from Baseline at Wk 48	0.30 pounds Standard Deviation 3.353
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry L Hand Grip, Change from Baseline at Wk 60	1.04 pounds Standard Deviation 4.811
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Hand Grip, Baseline	17.17 pounds Standard Deviation 7.266
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Hand Grip, Change from Baseline at Wk 48	-0.79 pounds Standard Deviation 3.628
Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, Shoulder Abduction, and Hand Grip as Assessed by Myometry R Hand Grip, Change from Baseline at Wk 60	-0.61 pounds Standard Deviation 3.010

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable heart rate data. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

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 rate values were collected before, during, and after the 6MWT. The participant rested for 5 minutes in a sitting position before the 6MWT, and the mean heart rate for the last minute of this rest period was obtained 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 obtained and documented as the recovery heart rate.

Outcome measures

Outcome measures
Measure	Ataluren n=24 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Resting Heart Rate, Baseline	106.46 beeps per minute Standard Deviation 11.163
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Resting Heart Rate, Change from Baseline at Wk 48	8.30 beeps per minute Standard Deviation 12.382
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Resting Heart Rate, Change from Baseline at Wk 60	5.16 beeps per minute Standard Deviation 11.197
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Active Heart Rate, Baseline	152.17 beeps per minute Standard Deviation 13.457
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Active Heart Rate, Change from Baseline at Wk 48	3.86 beeps per minute Standard Deviation 12.365
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Active Heart Rate, Change from Baseline at Wk 60	-2.84 beeps per minute Standard Deviation 17.238
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Recovery Heart Rate, Baseline	113.13 beeps per minute Standard Deviation 12.664
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Recovery Heart Rate, Change from Baseline at Wk 48	4.64 beeps per minute Standard Deviation 9.079
Change in Resting, Active, and Recovery Heart Rate as Assessed by Heart Rate Monitoring With the Polar RS400 Recovery Heart Rate, Change from Baseline at Wk 60	1.68 beeps per minute Standard Deviation 15.688

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable verbal memory and attention data.

The digit span task is a 2-part (forward and backward) test in which a series of digits (3 to 9) were presented to participant in an auditory format only. For forward condition, the participant was to repeat digits back in the order they were presented. For backward condition, the participant was to reverse the order of presentation. Maximum score for each part (digit forward and digit backward) of task is 14; participants received a score of 2 points if they passed both trials, score of 1 point if they passed only 1 trial, and score of 0 points if they failed both trials. A raw score of total number of correct forward and backward responses was age-normalized by subtracting corresponding mean and dividing by corresponding standard deviation of a reference population for that age. For each forward and backward result, the resulting Z score was transformed into percentile rank of normal distribution. Change from Baseline in number of digits recalled forward and backward is reported.

Outcome measures

Outcome measures
Measure	Ataluren n=35 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Forward, Baseline	3.66 digits recalled Standard Deviation 2.378
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Forward, Change from Baseline at Wk 48	0.14 digits recalled Standard Deviation 1.353
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Forward, Change from Baseline at Wk 60	0.04 digits recalled Standard Deviation 1.453
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Backward, Baseline	3.15 digits recalled Standard Deviation 2.524
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Backward, Change from Baseline at Wk 48	0.74 digits recalled Standard Deviation 1.442
Change From Baseline in Verbal Memory and Attention as Assessed by the Digit Span Task Recalled Backward, Change from Baseline at Wk 60	0.74 digits recalled Standard Deviation 1.442

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable participant-reported PedsQL data.

The PedsQL Generic Core Scales comprises 23 questions, which are grouped into 4 scales (physical functioning, emotional functioning, social functioning, and school functioning); the fatigue-specific module included an additional 18 questions. The appropriate age-specific version was completed. On PedsQL Generic Core Scales, items were reversed scored and linearly transformed to a 0 to 100 scale so that higher scores indicate a better HRQL (0=100, 1=75, 2=50, 3=25, and 4=0). Mean is the sum of the items over the number of items that were answered (which accounts for missing data) to create scale scores. If \>50% of the items in a scale were missing, the scale score was not computed. Mean Total Scale Score is the sum of all of the items over the number of items that were answered on all scales.

Outcome measures

Outcome measures
Measure	Ataluren n=35 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Physical Functioning, Baseline	45.13 scores on a scale Standard Deviation 21.114
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Physical Functioning Change from Baseline at Wk 48	3.16 scores on a scale Standard Deviation 20.180
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Physical Functioning Change from Baseline at Wk 60	6.25 scores on a scale Standard Deviation 23.545
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Emotional Functioning, Baseline	65.29 scores on a scale Standard Deviation 16.141
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Emotional Function, Change from Baseline at Wk 48	8.24 scores on a scale Standard Deviation 18.294
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Emotional Function, Change from Baseline at Wk 60	12.22 scores on a scale Standard Deviation 17.614
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Social Functioning, Baseline	61.80 scores on a scale Standard Deviation 16.914
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Social Functioning, Change from Baseline at Wk 48	4.82 scores on a scale Standard Deviation 19.463
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Social Functioning, Change from Baseline at Wk 60	3.75 scores on a scale Standard Deviation 15.861
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory School Functioning, Baseline	70.29 scores on a scale Standard Deviation 16.466
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory School Functioning, Change from Baseline at Wk 48	1.76 scores on a scale Standard Deviation 17.917
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory School Functioning, Change from Baseline at Wk 60	3.29 scores on a scale Standard Deviation 16.273
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Fatigue Scale Score, Baseline	74.28 scores on a scale Standard Deviation 14.917
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Fatigue Scale Score, Change from Baseline at Wk 48	5.08 scores on a scale Standard Deviation 12.661
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Fatigue Scale Score, Change from Baseline at Wk 60	4.39 scores on a scale Standard Deviation 11.777
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Total Score, Baseline	58.59 scores on a scale Standard Deviation 12.793
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Total Score, Change from Baseline at Wk 48	4.28 scores on a scale Standard Deviation 13.406
Change From Baseline in Participant-Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Total Score, Change from Baseline at Wk 60	6.42 scores on a scale Standard Deviation 10.887

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable parent- or caregiver-reported PedsQL data.

The PedsQL Generic Core Scales comprises 23 questions, which are grouped into 4 scales (physical functioning, emotional functioning, social functioning, and school functioning); the fatigue-specific module included an additional 18 questions. The appropriate age-specific version was completed. On PedsQL Generic Core Scales, items were reversed scored and linearly transformed to a 0 to 100 scale so that higher scores indicate a better HRQL (0=100, 1=75, 2=50, 3=25, and 4=0). Mean is the sum of the items over the number of items that were answered (which accounts for missing data) to create scale scores. If \>50% of the items in a scale were missing, the scale score was not computed. Mean Fatigue Scale Score is the sum of the items over the number of items that were answered in the Emotional, Social, and School Functioning Scales. Mean Total Scale Score is the sum of all of the items over the number of items that were answered on all scales.

Outcome measures

Outcome measures
Measure	Ataluren n=35 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Physical Functioning Change from Baseline at Wk 60	1.85 scores on a scale Standard Deviation 19.366
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Social Functioning, Change from Baseline at Wk 60	6.16 scores on a scale Standard Deviation 17.599
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Physical Functioning, Baseline	48.07 scores on a scale Standard Deviation 21.111
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Physical Functioning Change from Baseline at Wk 48	2.31 scores on a scale Standard Deviation 20.284
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Emotional Functioning, Baseline	69.45 scores on a scale Standard Deviation 17.465
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Emotional Function, Change from Baseline at Wk 48	5.27 scores on a scale Standard Deviation 19.595
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Emotional Function, Change from Baseline at Wk 60	3.29 scores on a scale Standard Deviation 20.021
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Social Functioning, Baseline	57.95 scores on a scale Standard Deviation 19.397
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Social Functioning, Change from Baseline at Wk 48	9.38 scores on a scale Standard Deviation 16.956
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory School Functioning, Baseline	67.61 scores on a scale Standard Deviation 14.944
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory School Functioning, Change from Baseline at Wk 48	5.66 scores on a scale Standard Deviation 15.824
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory School Functioning, Change from Baseline at Wk 60	-0.37 scores on a scale Standard Deviation 10.651
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Fatigue Scale Score, Baseline	72.25 scores on a scale Standard Deviation 13.777
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Fatigue Scale Score, Change from Baseline at Wk 48	0.77 scores on a scale Standard Deviation 9.703
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Fatigue Scale Score, Change from Baseline at Wk 60	-0.40 scores on a scale Standard Deviation 9.269
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Total Score, Baseline	59.08 scores on a scale Standard Deviation 13.348
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Total Score, Change from Baseline at Wk 48	5.24 scores on a scale Standard Deviation 11.409
Change From Baseline in Parent- or Caregiver-Reported HRQL as Measured by the PedsQL Inventory Total Score, Change from Baseline at Wk 60	2.68 scores on a scale Standard Deviation 12.261

SECONDARY outcome

Timeframe: Baseline, Week 48 and Week 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable CK data.

Serum CK concentrations (as measured by the central laboratory) were quantified from the blood samples that were collected as part of the safety laboratory evaluations. The normal range for CK is 18 to 363 units/liter (U/L).

Outcome measures

Outcome measures
Measure	Ataluren n=35 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Serum Creatine Kinase (CK) Levels Change from Baseline at Week 60	-549.57 U/L Standard Deviation 3261.151
Change From Baseline in Serum Creatine Kinase (CK) Levels Baseline	5988.31 U/L Standard Deviation 3967.714
Change From Baseline in Serum Creatine Kinase (CK) Levels Change from Baseline at Week 48	84.06 U/L Standard Deviation 2641.422

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: All enrolled participants who received at least 1 dose of study drug (As-Treated Population) and had evaluable dystrophin expression data.

The biceps muscle was biopsied from 1 arm for confirmation of the absence or low levels of dystrophin prior to treatment initiation and from the other arm to assess for production of dystrophin post-treatment. Muscle tissue sections were processed and immunostained to detect muscle membrane-localized dystrophin. An increase in value indicates dystrophin production.

Outcome measures

Outcome measures
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Change From Baseline in Dystrophin Expression on Biceps Muscle Biopsy as Measured by Immunofluorescence Staining of the Sarcolemmal Membrane With an Antibody to the C-Terminal Portion of the Dystrophin Protein Baseline	16.828 dystrophin positive fibers Standard Deviation 8.4285
Change From Baseline in Dystrophin Expression on Biceps Muscle Biopsy as Measured by Immunofluorescence Staining of the Sarcolemmal Membrane With an Antibody to the C-Terminal Portion of the Dystrophin Protein Change from Baseline	-1.880 dystrophin positive fibers Standard Deviation 5.1337

SECONDARY outcome

Timeframe: Baseline up to Week 89

Population: All enrolled participants who received at least 1 dose of study drug (As-Treated Population).

Study drug compliance was assessed by the participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the amount of drug actually taken relative to the amount that was prescribed. Physician-prescribed dose reductions and interruptions were factored into the calculations. "Not recorded" applies only to the days on which all dosing information was missing or for missing days. Invalid entries in the participant daily diary were assigned values of 0.0 for percentage of doses taken and 99.0 for percentage of doses not recorded.

Outcome measures

Outcome measures
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Study Drug Compliance Percentage of Doses Taken as Planned	84.7 percentage of doses of study drug Interval 0.0 to 99.2
Study Drug Compliance Percentage of Doses Missed	1.1 percentage of doses of study drug Interval 0.0 to 13.2
Study Drug Compliance Percentage of Doses Changed	0.3 percentage of doses of study drug Interval 0.0 to 22.2
Study Drug Compliance Percentage of Doses Not Recorded	10.2 percentage of doses of study drug Interval 0.0 to 99.0

SECONDARY outcome

Timeframe: 0 (pre-dose), 0.5, 1, 2, 3, and 4 hours post-morning dose and 0 (pre-dose) and 2 hours post-midday dose on Day 2 of Week 1 and Day 2 of Week 6

Population: All enrolled participants who received at least 1 dose of study drug (As-Treated Population) and had evaluable plasma data.

Blood for ataluren concentrations over a 24-hour period was to be collected on Day 2 of Week 1 and Day 2 of Week 6. Analysis of the blood samples was to be conducted 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/milliliters (μg/mL). Plasma concentrations below qualification (BQ) is treated as 0 in the summary calculation.

Outcome measures

Outcome measures
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Pharmacokinetics: Ataluren Plasma Exposure in All Participants Pre-Morning Dose, Week 1	0 μg/mL Standard Deviation 0
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 30 minutes Post-Morning Dose, Week 1	14.59 μg/mL Standard Deviation 10.404
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 1 hour Post-Morning Dose, Week 1	16.24 μg/mL Standard Deviation 10.251
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 2 hours Post-Morning Dose, Week 1	23.71 μg/mL Standard Deviation 12.895
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 3 hours Post-Dose, Week 1	28.96 μg/mL Standard Deviation 15.306
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 4 hours Post-Morning Dose, Week 1	29.44 μg/mL Standard Deviation 14.854
Pharmacokinetics: Ataluren Plasma Exposure in All Participants Pre-Midday Dose, Week 1	20.43 μg/mL Standard Deviation 15.256
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 2 hours Post-Midday Dose, Week 1	35.00 μg/mL Standard Deviation 17.172
Pharmacokinetics: Ataluren Plasma Exposure in All Participants Pre-Morning Dose, Week 6	19.99 μg/mL Standard Deviation 15.598
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 30 minutes Post-Morning Dose, Week 6	24.50 μg/mL Standard Deviation 17.916
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 1 hour Post-Morning Dose, Week 6	24.68 μg/mL Standard Deviation 19.577
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 2 hours Post-Morning Dose, Week 6	26.79 μg/mL Standard Deviation 18.709
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 3 hours Post-Morning Dose, Week 6	26.40 μg/mL Standard Deviation 16.961
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 4 hours Post-Morning Dose, Week 6	24.04 μg/mL Standard Deviation 17.641
Pharmacokinetics: Ataluren Plasma Exposure in All Participants Pre-Midday Dose, Week 6	17.15 μg/mL Standard Deviation 13.190
Pharmacokinetics: Ataluren Plasma Exposure in All Participants 2 hours Post-Midday Dose, Week 6	25.49 μg/mL Standard Deviation 14.297

SECONDARY outcome

Timeframe: 0 (pre-dose), 0.5, 1, 2, 3, and 4 hours post-morning dose and 0 (pre-dose) and 2 hours post-midday dose on Day 2 of Week 1 and Day 2 of Week 6

Population: All enrolled participants who received at least 1 dose of study drug, were ambulatory, and had evaluable plasma data. Participants who were ambulatory were able to walk ≥75 meters unassisted at screening.

Blood for ataluren concentrations were collected on Day 2 of Week 1 and Day 2 of Week 6. Analysis of the blood samples was to be conducted using a validated HPLC-MS/MS method with a LLOQ of 0.5 μg/mL. Plasma concentrations BQ is treated as 0 in the summary calculation.

Outcome measures

Outcome measures
Measure	Ataluren n=25 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants Pre-Morning Dose, Week 1	0 μg/mL Standard Deviation 0
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 30 minutes Post-Morning Dose, Week 1	14.88 μg/mL Standard Deviation 10.771
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 1 hour Post-Morning Dose, Week 1	14.65 μg/mL Standard Deviation 9.608
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 2 hours Post-Morning Dose, Week 1	20.96 μg/mL Standard Deviation 12.648
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 3 hours Post-Dose, Week 1	26.29 μg/mL Standard Deviation 15.853
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 4 hours Post-Morning Dose, Week 1	26.19 μg/mL Standard Deviation 14.531
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants Pre-Midday Dose, Week 1	15.26 μg/mL Standard Deviation 10.238
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 2 hours Post-Midday Dose, Week 1	32.08 μg/mL Standard Deviation 17.977
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants Pre-Morning Dose, Week 6	14.83 μg/mL Standard Deviation 10.032
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 30 minutes Post-Morning Dose, Week 6	18.08 μg/mL Standard Deviation 8.654
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 1 hour Post-Morning Dose, Week 6	17.75 μg/mL Standard Deviation 8.660
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 2 hours Post-Morning Dose, Week 6	20.33 μg/mL Standard Deviation 9.103
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 3 hours Post-Morning Dose, Week 6	20.13 μg/mL Standard Deviation 9.423
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 4 hours Post-Morning Dose, Week 6	18.72 μg/mL Standard Deviation 12.255
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants Pre-Midday Dose, Week 6	12.40 μg/mL Standard Deviation 6.775
Pharmacokinetics: Ataluren Plasma Exposure in Ambulatory Participants 2 hours Post-Midday Dose, Week 6	21.09 μg/mL Standard Deviation 7.959

SECONDARY outcome

Timeframe: 0 (pre-dose), 0.5, 1, 2, 3, and 4 hours post-morning dose and 0 (pre-dose) and 2 hours post-midday dose on Day 2 of Week 1 and Day 2 of Week 6

Population: All enrolled participants who received at least 1 dose of study drug (As-Treated Population) and had evaluable prednisone or deflazacort plasma data.

Blood for prednisone and deflazacort concentrations were collected on Day 2 of Week 1 and Day 2 of Week 6. Plasma samples for the determination of prednisone concentrations were analyzed using a validated HPLC/MS/MS method, with LLOQs of 1.00 nanograms/milliliters (ng/mL). Prednisone concentrations \<1.01 are treated as 1.01 in the summary calculation. Plasma samples for the determination of 21-desacetyl deflazacort concentrations were analyzed using a validated HPLC/MS/MS method with an LLOQ of 1.0 ng/mL. Deflazacort concentrations BQ are treated as 0 in the summary calculation.

Outcome measures

Outcome measures
Measure	Ataluren n=36 Participants Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 8 hours Post-Dose, Prednisone, Week 6	13.52 ng/mL Standard Deviation 11.872
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort Pre-Dose, Deflazacort, Week 1	0 ng/mL Standard Deviation 0
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 30 minutes Post-Dose, Deflazacort, Week 1	28.25 ng/mL Standard Deviation 31.968
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 1 hours Post-Dose, Deflazacort, Week 1	49.47 ng/mL Standard Deviation 43.631
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 2 hours Post-Dose, Deflazacort, Week 1	74.00 ng/mL Standard Deviation 49.349
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 3 hours Post-Dose, Deflazacort, Week 1	63.34 ng/mL Standard Deviation 38.045
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 4 hours Post-Dose, Deflazacort, Week 1	45.50 ng/mL Standard Deviation 31.427
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort Pre-Dose, Prednisone, Week 1	1.01 ng/mL Standard Deviation 0
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 30 minutes Post-Dose, Prednisone, Week 1	18.27 ng/mL Standard Deviation 14.451
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 1 hour Post-Dose, Prednisone, Week 1	35.48 ng/mL Standard Deviation 24.026
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 2 hours Post-Dose, Prednisone, Week 1	32.41 ng/mL Standard Deviation 27.603
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 3 hours Post-Dose, Prednisone, Week 1	57.12 ng/mL Standard Deviation 6.948
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 4 hours Post-Dose, Prednisone, Week 1	49.44 ng/mL Standard Deviation 12.139
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 6 hours Post-Dose, Prednisone, Week 1	31.41 ng/mL Standard Deviation 12.367
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 8 hours Post-Dose, Prednisone, Week 1	15.59 ng/mL Standard Deviation 8.735
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort Pre-Dose, Prednisone, Week 6	1.01 ng/mL Standard Deviation 0
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 30 minutes Post-Dose, Prednisone, Week 6	17.13 ng/mL Standard Deviation 16.376
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 1 hour Post-Dose, Prednisone, Week 6	32.48 ng/mL Standard Deviation 21.190
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 2 hours Post-Dose, Prednisone, Week 6	39.91 ng/mL Standard Deviation 26.374
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 3 hours Post-Dose, Prednisone, Week 6	41.02 ng/mL Standard Deviation 13.444
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 4 hours Post-Dose, Prednisone, Week 6	38.82 ng/mL Standard Deviation 5.934
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 6 hours Post-Dose, Prednisone, Week 6	24.95 ng/mL Standard Deviation 14.243
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 6 hours Post-Dose, Deflazacort, Week 1	18.67 ng/mL Standard Deviation 17.648
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 8 hours Post-Dose, Deflazacort, Week 1	7.57 ng/mL Standard Deviation 9.418
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort Pre-Dose, Deflazacort, Week 6	0.06 ng/mL Standard Deviation 0.268
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 30 minutes Post-Dose, Deflazacort, Week 6	33.46 ng/mL Standard Deviation 32.511
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 1 hour Post-Dose, Deflazacort, Week 6	71.94 ng/mL Standard Deviation 77.370
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 2 hours Post-Dose, Deflazacort, Week 6	81.41 ng/mL Standard Deviation 44.945
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 3 hours Post-Dose, Deflazacort, Week 6	49.63 ng/mL Standard Deviation 26.633
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 4 hours Post-Dose, Deflazacort, Week 6	25.85 ng/mL Standard Deviation 16.126
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 6 hours Post-Dose, Deflazacort, Week 6	10.14 ng/mL Standard Deviation 14.080
Corticosteroid Plasma Concentrations as Assessed by a Validated Bioanalytical Method, in Participants Who Received a Daily Corticosteroid Regimen With Prednisone or Deflazacort 8 hours Post-Dose, Deflazacort, Week 6	4.15 ng/mL Standard Deviation 7.985

SECONDARY outcome

Timeframe: Baseline, Week 48, Week 60

Population: All enrolled participants who received at least 1 dose of study drug and had Baseline and on-treatment data (Evaluable Population) with evaluable muscle composition data. Muscle composition data were not collected or analyzed for this extension study.

This Outcome Measure is an exploratory study objective and data were not collected or analyzed for this extension study.

Outcome measures

Outcome data not reported

Adverse Events

Ataluren

Serious events: 6 serious events

Other events: 36 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ataluren n=36 participants at risk Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Infections and infestations Influenza	2.8% 1/36 • Number of events 1 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Pneumonia	2.8% 1/36 • Number of events 1 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Femur fracture	2.8% 1/36 • Number of events 1 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Muscle contracture	2.8% 1/36 • Number of events 1 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Respiratory failure	2.8% 1/36 • Number of events 1 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Vascular disorders Hypertension	5.6% 2/36 • Number of events 2 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).

Other adverse events

Other adverse events
Measure	Ataluren n=36 participants at risk Participants received ataluren 3 times per day with meals at doses of 20 mg/kg (breakfast), 20 mg/kg (lunch), and 40 mg/kg (dinner) for up to 89 weeks.
Cardiac disorders Myocardial fibrosis	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Cardiac disorders Tachycardia	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Abdominal pain	16.7% 6/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Abdominal pain upper	30.6% 11/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Constipation	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Diarrhoea	22.2% 8/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Flatulence	33.3% 12/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Nausea	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Stomach discomfort	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Gastrointestinal disorders Vomiting	66.7% 24/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
General disorders Asthenia	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
General disorders Disease progression	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
General disorders Gait disturbance	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
General disorders Pyrexia	19.4% 7/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Bronchitis	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Gastroenteritis	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Gastroenteritis viral	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Influenza	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Localized Infections	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Nasopharyngitis	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Otitis media	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Rhinitis	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Sinusitis	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Infections and infestations Upper respiratory tract infection	27.8% 10/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Contusion	19.4% 7/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Excoriation	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Fall	30.6% 11/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Head injury	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Iliotibial band syndrome	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Joint injury	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Joint sprain	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Limb injury	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Postprocedural discomfort	25.0% 9/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Procedural pain	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Injury, poisoning and procedural complications Spinal compression fracture	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Investigations Weight decreased	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Metabolism and nutrition disorders Decreased appetite	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Metabolism and nutrition disorders Insulin resistance	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Arthralgia	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Back pain	16.7% 6/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Groin pain	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Joint contracture	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Muscular weakness	25.0% 9/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Musculoskeletal pain	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Myalgia	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 6/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Scoliosis	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Musculoskeletal and connective tissue disorders Tendinous contracture	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Nervous system disorders Headache	47.2% 17/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Nervous system disorders Migraine	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Nervous system disorders Motor dysfunction	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Renal and urinary disorders Haematuria	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Renal and urinary disorders Urinary incontinence	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Apnoea	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Cough	38.9% 14/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Dyspnoea	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Epistaxis	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Nasal congestion	30.6% 11/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	16.7% 6/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Respiratory tract congestion	11.1% 4/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Sinus congestion	13.9% 5/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Sleep apnoea syndrome	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Respiratory, thoracic and mediastinal disorders Upper respiratory tract congestion	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Skin and subcutaneous tissue disorders Dermatitis contact	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Skin and subcutaneous tissue disorders Erythema	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Skin and subcutaneous tissue disorders Rash	16.7% 6/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Skin and subcutaneous tissue disorders Urticaria	8.3% 3/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Vascular disorders Flushing	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).
Vascular disorders Hypertension	5.6% 2/36 • Baseline up to Week 89 All enrolled participants who received at least 1 dose of study drug (As-Treated Population).

Additional Information

Patient Advocacy

PTC Therapeutics, Inc.

Phone: 1-866-562-4620

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the Sponsor for review. The Sponsor may consult with the PI to require changes to the communication or extend the embargo.
Publication restrictions are in place

Restriction type: OTHER