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

• ClinicalTrials.gov Identifier: NCT00592553
• Recruitment Status: Completed
• First Posted: January 14, 2008
• Results First Posted: April 7, 2020
• Last Update Posted: April 7, 2020
• Sponsor: PTC Therapeutics
• Information provided by (Responsible Party): PTC Therapeutics

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
• Interventions: Drug: Ataluren; Drug: Placebo
• Enrollment: 174

Participant Flow

Recruitment Details	A total of 185 participants were screened for eligibility, of which 11 participants did not meet entry criteria.
Pre-assignment Details	A total of 174 eligible participants were randomized in 1:1:1 ratio to receive either placebo, low-dose ataluren, or high-dose ataluren.

Arm/Group Title	High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description	Participants received 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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Period Title: Overall Study
Started	60	57	57
As-treated Population [1]	60	57	57
ITT Population [2]	60	57	57
Completed	59	57	57
Not Completed	1	0	0
Reason Not Completed
Protocol Noncompliance	1	0	0
[1] All Randomized participants who received any study treatment.; [2] Received any study treatment; had a valid baseline, and at least 1 valid post-baseline 6MWD value.

Baseline Characteristics

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo	Total
Arm/Group Description		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.	Total of all reporting groups
Overall Number of Baseline Participants		60	57	57	174
Baseline Analysis Population Description		As-treated population included all randomized participants who actually received any study treatment.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	60 participants	57 participants	57 participants	174 participants
		8.4 (2.53)	8.8 (2.91)	8.3 (2.33)	8.5 (2.59)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	60 participants	57 participants	57 participants	174 participants
	Female	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Male	60 100.0%	57 100.0%	57 100.0%	174 100.0%
6-Minute Walk Distance (6MWD) [1]; Mean (Standard Deviation); Unit of measure: Meters
	Number Analyzed	60 participants	57 participants	57 participants	174 participants
		358.2 (103.97)	350.0 (97.55)	359.6 (87.67)	356.0 (96.30)
		[1] Measure 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.

Outcome Measures

1. Primary Outcome

Title	Change From Baseline in 6MWD at Week 48
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

Outcome Measure Data

Analysis Population Description

Intent-to-treat (ITT) population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.

Arm/Group Title	High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:	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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed	59	55	55
Mean (Standard Deviation); Unit of Measure: meters
	-41.81 (89.234)	-12.86 (72.007)	-42.56 (90.046)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	High-Dose Ataluren, Placebo
	Comments	Analysis was performed using mixed model for repeated measures (MMRM) method including rank transformed 6MWD as the dependent variable; and rank transformed baseline 6MWD, treatment, visit, age (less than [<] 9 years versus [vs.] greater than or equal to [>=] 9 years) and corticosteroid use (yes vs. no) stratification factors, and interaction between treatment and visit as independent variables.
	Type of Statistical Test	Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.4756
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	Least Square (LS) Mean Difference
	Estimated Value	-30.52
	Confidence Interval	(2-Sided) 95%; -114.8 to 53.75
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 42.68
	Estimation Comments	[Not Specified]

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Low-Dose Ataluren, Placebo
	Comments	Analysis was performed using MMRM method including rank transformed 6MWD as the dependent variable; and rank transformed baseline 6MWD, treatment, visit, age (< 9 years vs. >= 9 years) and corticosteroid use (yes vs. no) stratification factors, and interaction between treatment and visit as independent variables.
	Type of Statistical Test	Other
	Comments	[Not Specified]
Statistical Test of Hypothesis	P-Value	0.1490
	Comments	[Not Specified]
	Method	Mixed Models Analysis
	Comments	[Not Specified]
Method of Estimation	Estimation Parameter	LS Mean Difference
	Estimated Value	62.65
	Confidence Interval	(2-Sided) 95%; -22.66 to 147.96
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 43.21
	Estimation Comments	[Not Specified]

2. Secondary Outcome

Title	Change From Baseline in Time to Stand From Supine Position at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: seconds
Baseline	Number Analyzed	60 participants	57 participants	57 participants
		12.25 (11.191)	10.80 (9.924)	11.50 (11.440)
Change at Week 48	Number Analyzed	59 participants	57 participants	56 participants
		3.00 (5.686)	3.23 (5.761)	3.24 (7.253)

3. Secondary Outcome

Title	Change From Baseline in Time to Walk/Run 10 Meters at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: seconds
Baseline	Number Analyzed	60 participants	57 participants	57 participants
		7.80 (5.243)	7.45 (4.373)	6.86 (2.813)
Change at Week 48	Number Analyzed	59 participants	57 participants	56 participants
		2.37 (6.149)	1.68 (5.617)	3.03 (6.691)

4. Secondary Outcome

Title	Change From Baseline in Time to Climb 4 Stairs at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: seconds
Baseline	Number Analyzed	60 participants	57 participants	57 participants
		7.63 (7.522)	6.94 (6.474)	6.04 (5.661)
Change at Week 48	Number Analyzed	59 participants	57 participants	56 participants
		3.51 (6.794)	2.39 (4.618)	4.79 (7.949)

5. Secondary Outcome

Title	Change From Baseline in Time to Descend 4 Stairs at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: seconds
Baseline	Number Analyzed	60 participants	57 participants	57 participants
		6.75 (7.219)	6.08 (5.985)	5.52 (5.753)
Change at Week 48	Number Analyzed	59 participants	57 participants	56 participants
		2.95 (7.323)	2.41 (6.162)	4.03 (7.828)

6. Secondary Outcome

Title	Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, and Shoulder Abduction at Week 48, as Assessed by Myometry
Description	Upper and lower extremity myometry was performed using a myometer following standardized procedures. Muscle groups evaluated included knee flexors, knee extensors, elbow flexors, elbow extensors, and shoulder abductors. Bilateral assessments were done and 3 measurements were recorded from each muscle group on each side if possible. Mean values for the left and right sides were calculated.
Time Frame	Baseline, Week 48

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: pounds
Baseline: Force during knee flexion	Number Analyzed	60 participants	57 participants	57 participants
		12.45 (4.684)	12.08 (4.217)	11.06 (3.494)
Change at Week 48: Force during knee flexion	Number Analyzed	59 participants	57 participants	55 participants
		0.39 (3.148)	-0.07 (3.511)	0.38 (2.991)
Baseline: Force during knee extension	Number Analyzed	60 participants	57 participants	56 participants
		12.71 (7.910)	12.81 (5.753)	12.96 (6.162)
Change at Week 48: Force during knee extension	Number Analyzed	59 participants	57 participants	55 participants
		-0.59 (3.511)	-0.63 (3.616)	-1.85 (3.899)
Baseline: Force exerted during elbow flexion	Number Analyzed	60 participants	57 participants	57 participants
		8.72 (4.709)	7.66 (3.154)	8.14 (2.972)
Change at Week 48: Force during elbow flexion	Number Analyzed	59 participants	57 participants	56 participants
		-0.50 (1.832)	-0.10 (1.680)	-0.35 (1.807)
Baseline: Force during elbow extension	Number Analyzed	60 participants	57 participants	57 participants
		6.81 (3.815)	6.19 (3.083)	6.77 (2.785)
Change at Week 48: Force during elbow extension	Number Analyzed	59 participants	57 participants	56 participants
		-0.28 (1.473)	0.10 (1.493)	-0.51 (2.333)

7. Secondary Outcome

Title	Change From Baseline in Mean Activity Period/Day/Visit at Week 48, as Assessed by Step Activity Monitoring (SAM)
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. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. 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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: minutes
Baseline	Number Analyzed	59 participants	53 participants	54 participants
		756.84 (84.612)	761.86 (76.984)	751.71 (60.513)
Change at Week 48	Number Analyzed	56 participants	53 participants	49 participants
		0.87 (57.833)	-22.23 (84.759)	-19.91 (91.960)

8. Secondary Outcome

Title	Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 48, as Assessed by SAM
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. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. 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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: steps/day
Baseline	Number Analyzed	59 participants	53 participants	54 participants
		5302.31 (1907.058)	4870.13 (2165.522)	5602.31 (2023.543)
Change at Week 48	Number Analyzed	56 participants	53 participants	49 participants
		-615.14 (1468.452)	-676.46 (1717.535)	-908.34 (1999.969)

9. Secondary Outcome

Title	Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 48, as Assessed by SAM
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. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. 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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: steps/hour
Baseline	Number Analyzed	59 participants	53 participants	54 participants
		423.67 (168.754)	383.62 (161.911)	446.37 (160.666)
Change at Week 48	Number Analyzed	56 participants	53 participants	49 participants
		-44.51 (125.154)	-42.23 (126.429)	-59.62 (153.054)

10. Secondary Outcome

Title	Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 48, as Assessed by SAM
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. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. 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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: steps
Baseline: 10-minute total step count	Number Analyzed	59 participants	55 participants	54 participants
		35.77 (10.222)	32.24 (11.374)	36.76 (8.935)
Change at Week 48: 10-minute total step count	Number Analyzed	56 participants	54 participants	49 participants
		-2.77 (8.569)	-2.79 (6.355)	-3.97 (9.720)
Baseline: 20-minute total step count	Number Analyzed	59 participants	55 participants	54 participants
		29.13 (9.272)	25.68 (10.038)	29.74 (8.205)
Change at Week 48: 20-minute total step count	Number Analyzed	56 participants	54 participants	49 participants
		-2.49 (7.407)	-2.40 (5.806)	-3.55 (8.677)
Baseline: 30-minute total step count	Number Analyzed	59 participants	55 participants	54 participants
		25.00 (8.053)	22.08 (9.206)	25.70 (7.350)
Change at Week 48: 30-minute total step count	Number Analyzed	56 participants	54 participants	49 participants
		-2.08 (6.519)	-2.31 (5.505)	-3.03 (7.604)
Baseline: 60-minute total step count	Number Analyzed	59 participants	55 participants	54 participants
		18.58 (6.210)	16.52 (7.199)	19.50 (5.887)
Change at Week 48: 60-minute total step count	Number Analyzed	56 participants	54 participants	49 participants
		-1.50 (5.177)	-1.85 (4.616)	-2.33 (6.241)

11. Secondary Outcome

Title	Change From Baseline in Percentage of Time During the Active Period Spent at Low Activity (Less Than or Equal to [≤] 15 Steps/Minute), Medium Activity (16-30 Steps/Minute), and High Activity (Greater Than [>]30 Steps/Minute) at Week 48
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. Proportion of time during active periods spent at low activity(≤15 steps/minute), medium activity(16-30 steps/minute), and high activity(>30 steps/minute) were computed for each participant. Mean obtained during Screening and following Week 1 visit were used as baseline data for analysis. 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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: percentage of time
Baseline: Time spent at low activity	Number Analyzed	59 participants	53 participants	54 participants
		32.91 (7.842)	32.38 (8.213)	32.86 (6.239)
Change at Week 48: Time spent at low activity	Number Analyzed	56 participants	53 participants	49 participants
		-2.06 (7.903)	-1.12 (8.220)	-1.11 (5.586)
Baseline: Time spent at medium activity	Number Analyzed	59 participants	53 participants	54 participants
		11.11 (4.013)	10.00 (3.656)	11.84 (4.304)
Change at Week 48: Time spent at medium activity	Number Analyzed	56 participants	53 participants	49 participants
		-1.35 (3.348)	-0.69 (3.828)	-1.92 (4.178)
Baseline: Time spent at high activity	Number Analyzed	59 participants	53 participants	54 participants
		6.59 (4.077)	5.78 (3.785)	7.17 (3.700)
Change at Week 48: Time spent at high activity	Number Analyzed	54 participants	52 participants	48 participants
		-0.66 (2.790)	-0.96 (2.828)	-1.03 (3.783)

12. 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 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units on a scale
Baseline: Physical functioning score	Number Analyzed	58 participants	56 participants	56 participants
		63.63 (20.029)	59.27 (22.782)	61.87 (19.411)
Change at Week 48: Physical functioning score	Number Analyzed	54 participants	55 participants	50 participants
		-0.94 (19.125)	2.37 (25.105)	-1.00 (24.022)
Baseline: Emotional functioning score	Number Analyzed	58 participants	55 participants	56 participants
		73.92 (20.418)	73.70 (20.223)	70.13 (19.332)
Change at Week 48: Emotional functioning score	Number Analyzed	54 participants	54 participants	50 participants
		2.36 (16.742)	-1.83 (23.725)	4.30 (22.315)
Baseline: Social functioning score	Number Analyzed	58 participants	55 participants	55 participants
		67.50 (21.749)	65.09 (18.421)	63.36 (20.476)
Change at Week 48: Social functioning score	Number Analyzed	54 participants	54 participants	51 participants
		5.37 (20.463)	3.89 (21.841)	7.75 (18.870)
Baseline: School functioning score	Number Analyzed	58 participants	55 participants	54 participants
		67.72 (19.276)	64.55 (20.396)	64.65 (17.841)
Change at Week 48: School functioning score	Number Analyzed	52 participants	54 participants	49 participants
		3.61 (13.008)	6.11 (23.765)	4.06 (23.244)

13. 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 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units on a scale
Baseline: Physical functioning score	Number Analyzed	60 participants	56 participants	57 participants
		56.15 (19.955)	54.96 (20.592)	51.47 (19.274)
Change at Week 48: Physical functioning score	Number Analyzed	59 participants	56 participants	57 participants
		0.03 (17.088)	-3.10 (16.550)	0.23 (23.712)
Baseline: Emotional functioning score	Number Analyzed	60 participants	56 participants	57 participants
		70.08 (16.836)	69.11 (18.711)	65.96 (17.964)
Change at Week 48: Emotional functioning score	Number Analyzed	59 participants	56 participants	57 participants
		4.07 (15.382)	3.39 (18.068)	1.21 (17.794)
Baseline: Social functioning score	Number Analyzed	60 participants	56 participants	57 participants
		61.58 (15.825)	62.77 (16.540)	55.79 (18.269)
Change at Week 48: Social functioning score	Number Analyzed	59 participants	56 participants	57 participants
		-0.40 (18.470)	-1.09 (14.268)	3.71 (14.130)
Baseline: School functioning score	Number Analyzed	60 participants	56 participants	57 participants
		66.17 (18.258)	66.16 (16.320)	61.93 (13.587)
Change at Week 48: School functioning score	Number Analyzed	58 participants	56 participants	56 participants
		2.73 (18.490)	-2.32 (15.462)	3.48 (13.913)

14. Secondary Outcome

Title	Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 48
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 48.
Time Frame	Baseline, Week 48

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units on a scale
Baseline	Number Analyzed	58 participants	55 participants	54 participants
		69.47 (16.525)	71.62 (16.474)	69.70 (15.263)
Change at Week 48	Number Analyzed	55 participants	54 participants	52 participants
		6.95 (13.460)	0.45 (23.068)	3.92 (16.512)

15. Secondary Outcome

Title	Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 48
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 48.
Time Frame	Baseline, Week 48

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units on a scale
Baseline	Number Analyzed	60 participants	56 participants	57 participants
		73.39 (13.671)	70.71 (12.720)	68.27 (13.170)
Change at Week 48	Number Analyzed	58 participants	54 participants	57 participants
		1.97 (13.873)	1.27 (12.095)	2.51 (12.039)

16. Secondary Outcome

Title	Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score
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	Week 48

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units on a scale
Effectiveness score	Number Analyzed	53 participants	55 participants	53 participants
		55.97 (27.796)	54.60 (22.307)	51.26 (23.536)
Side-effects score	Number Analyzed	55 participants	56 participants	55 participants
		96.36 (11.199)	97.77 (7.578)	96.89 (8.874)
Convenience score	Number Analyzed	57 participants	56 participants	55 participants
		55.85 (17.008)	58.23 (19.040)	60.91 (16.665)
Global satisfaction score	Number Analyzed	55 participants	56 participants	55 participants
		61.04 (25.967)	61.19 (23.691)	57.56 (21.851)

17. Secondary Outcome

Title	Change From Baseline in Participant/Caregiver-Reported Number of Daily Accidental Falls at Week 48
Description	Number of falls was determined by daily diary records maintained by participants and/or parent/caregivers.
Time Frame	Baseline, Week 48

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: falls/day
Baseline	Number Analyzed	54 participants	48 participants	48 participants
		0.40 (0.597)	0.27 (0.480)	0.54 (0.943)
Change at Week 48	Number Analyzed	52 participants	47 participants	44 participants
		-0.10 (0.466)	-0.06 (0.501)	0.20 (1.282)

18. Secondary Outcome

Title	Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: z-score
Baseline: Forward condition	Number Analyzed	59 participants	56 participants	55 participants
		3.59 (2.554)	2.89 (2.180)	2.84 (1.675)
Change at Week 48: Forward condition	Number Analyzed	57 participants	54 participants	52 participants
		0.39 (1.677)	0.50 (1.767)	0.40 (1.550)
Baseline: Backward condition	Number Analyzed	59 participants	56 participants	54 participants
		1.73 (1.846)	1.70 (1.868)	1.59 (1.460)
Change at Week 48: Backward condition	Number Analyzed	57 participants	54 participants	51 participants
		0.56 (1.500)	0.33 (1.441)	0.59 (1.203)

19. Secondary Outcome

Title	Change From Baseline in Heart Rate Before, During, and After Each 6MWT at Week 48, as Assessed by Heart Rate Monitoring With the Polar® RS400
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: beats/minute
Baseline: Resting heart rate	Number Analyzed	60 participants	57 participants	57 participants
		105.47 (12.908)	109.70 (9.908)	104.07 (11.588)
Change at Week 48: Resting heart rate	Number Analyzed	54 participants	53 participants	50 participants
		-0.63 (13.008)	-0.36 (10.223)	-0.26 (15.614)
Baseline: Active heart rate	Number Analyzed	60 participants	56 participants	57 participants
		142.67 (18.070)	141.77 (15.574)	136.67 (20.713)
Change at Week 48: Active heart rate	Number Analyzed	53 participants	49 participants	48 participants
		-5.00 (21.976)	2.39 (19.095)	1.65 (21.295)
Baseline: Recovery heart rate	Number Analyzed	60 participants	56 participants	57 participants
		109.90 (12.633)	113.48 (10.340)	107.86 (12.066)
Change at Week 48: Recovery heart rate	Number Analyzed	53 participants	49 participants	48 participants
		-0.23 (13.475)	-1.33 (13.270)	0.54 (15.181)

20. Secondary Outcome

Title	Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 48
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

Outcome Measure Data

Analysis Population Description

ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: units/liter (U/L)
Baseline	Number Analyzed	60 participants	57 participants	57 participants
		10853.65 (6251.136)	12084.70 (7772.631)	10569.60 (6488.477)
Change at Week 48	Number Analyzed	58 participants	57 participants	55 participants
		-1680.09 (4264.769)	-2146.32 (7151.944)	-1235.13 (4323.943)

21. Secondary Outcome

Title	Percent Change From Pre-Treatment Visit (1 Week Prior to Baseline Visit) in Biceps Muscle Dystrophin Expression at Post-Treatment Visit (Week 36), as Determined by Immunofluorescence
Description	Immunofluorescence evidence of a change in dystrophin expression on biceps muscle biopsy was defined as an increase in the staining of the sarcolemmal membrane with an antibody to the C-terminal portion of the dystrophin protein (excluding revertant fibers) between the pre-treatment (1 week prior to Baseline visit) and post-treatment (Week 36) biopsies. The biceps muscle was biopsied from one arm for confirmation of the absence or reduced levels of dystrophin prior to treatment initiation and from the other arm to assess for production of dystrophin post-treatment.
Time Frame	Pre-Treatment (1 week prior to baseline), post-treatment (Week 36)

Outcome Measure Data

Analysis Population Description

As-treated population included all randomized participants who actually received any study treatment. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.

Arm/Group Title		High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:		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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed		60	57	57
Mean (Standard Deviation); Unit of Measure: percent change
Pre-treatment	Number Analyzed	60 participants	55 participants	56 participants
		336.096 (138.9753)	359.797 (142.7361)	357.271 (139.6650)
Percent change post-treatment	Number Analyzed	59 participants	55 participants	56 participants
		-1.278 (27.7432)	-2.128 (28.8287)	-0.898 (19.2112)

22. Other Pre-specified Outcome

Title	Percentage of Participants With Treatment-Emergent Adverse Events (AEs)
Description	An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. Treatment-emergent adverse event (TEAE) was defined as an adverse event that occurred or worsened in the period extending from first dose of study drug to 6 weeks after the last dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
Time Frame	Baseline up to Week 54

Outcome Measure Data

Analysis Population Description

As-treated population included all randomized participants who actually received any study treatment.

Arm/Group Title	High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:	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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed	60	57	57
Measure Type: Number; Unit of Measure: percentage of participants
	95.0	96.5	98.2

23. Other Pre-specified Outcome

Title	Study Drug Compliance
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 to Week 48

Outcome Measure Data

Analysis Population Description

As-treated population included all randomized participants who actually received any study treatment.

Arm/Group Title	High-Dose Ataluren	Low-Dose Ataluren	Placebo
Arm/Group Description:	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 48 weeks.	Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.	Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
Overall Number of Participants Analyzed	60	57	57
Median (Full Range); Unit of Measure: percentage of drug
	97.87; (34.9 to 99.6)	97.03; (64.5 to 99.8)	97.74; (76.6 to 99.9)

Adverse Events

Time Frame	Baseline up to 6 weeks after the last dose of study drug (Week 54)
Adverse Event Reporting Description	As-treated population included all randomized participants who actually received any study treatment.
Arm/Group Title	High-Dose Ataluren		Low-Dose Ataluren		Placebo
Arm/Group Description	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 48 weeks.		Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 48 weeks.		Participants received placebo matched to ataluren orally TID at morning, midday, and evening for 48 weeks.
All-Cause Mortality
	High-Dose Ataluren		Low-Dose Ataluren		Placebo
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	--/--		--/--		--/--
Serious Adverse Events
	High-Dose Ataluren		Low-Dose Ataluren		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	2/60 (3.33%)		2/57 (3.51%)		3/57 (5.26%)
Cardiac disorders
Supraventricular tachycardia † 1	1/60 (1.67%)		0/57 (0.00%)		0/57 (0.00%)
Gastrointestinal disorders
Abdominal pain † 1	0/60 (0.00%)		0/57 (0.00%)		1/57 (1.75%)
Infections and infestations
Appendicitis † 1	0/60 (0.00%)		1/57 (1.75%)		0/57 (0.00%)
Influenza † 1	0/60 (0.00%)		0/57 (0.00%)		1/57 (1.75%)
Varicella † 1	0/60 (0.00%)		0/57 (0.00%)		1/57 (1.75%)
Injury, poisoning and procedural complications
Femur fracture † 1	0/60 (0.00%)		0/57 (0.00%)		1/57 (1.75%)
Lower limb fracture † 1	1/60 (1.67%)		0/57 (0.00%)		0/57 (0.00%)
Metabolism and nutrition disorders
Dehydration † 1	0/60 (0.00%)		1/57 (1.75%)		0/57 (0.00%)
Nervous system disorders
Grand mal convulsion † 1	0/60 (0.00%)		0/57 (0.00%)		1/57 (1.75%)
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 9.1
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	High-Dose Ataluren		Low-Dose Ataluren		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	58/60 (96.67%)		56/57 (98.25%)		57/57 (100.00%)
Blood and lymphatic system disorders
Lymphadenopathy † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	2/57 (3.51%)	2
Microcytic anaemia † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Cardiac disorders
Arrhythmia † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Dilatation ventricular † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Left ventricular hypertrophy † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Palpitations † 1	2/60 (3.33%)	2	1/57 (1.75%)	1	1/57 (1.75%)	1
Sinus bradycardia † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Supraventricular extrasystoles † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Supraventricular tachycardia † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Congenital, familial and genetic disorders
Kidney malformation † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Macroglossia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Ear and labyrinth disorders
Ear congestion † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	0/57 (0.00%)	0
Ear pain † 1	1/60 (1.67%)	1	2/57 (3.51%)	3	3/57 (5.26%)	3
Motion sickness † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Tinnitus † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	2
Tympanic membrane hyperaemia † 1	2/60 (3.33%)	2	2/57 (3.51%)	5	1/57 (1.75%)	1
Tympanic membrane perforation † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Endocrine disorders
Goitre † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Eye disorders
Abnormal sensation in eye † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Cataract † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Conjunctivitis † 1	3/60 (5.00%)	3	2/57 (3.51%)	2	1/57 (1.75%)	1
Conjunctivitis allergic † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Eye pain † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Eye pruritus † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Eye swelling † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Eyelid cyst † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Hypermetropia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Ocular hyperaemia † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Vision blurred † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Visual impairment † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Gastrointestinal disorders
Abdominal discomfort † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	4/57 (7.02%)	4
Abdominal pain † 1	12/60 (20.00%)	23	8/57 (14.04%)	8	4/57 (7.02%)	10
Abdominal pain lower † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Abdominal pain upper † 1	13/60 (21.67%)	32	9/57 (15.79%)	18	9/57 (15.79%)	13
Abdominal tenderness † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	1/57 (1.75%)	1
Aerophagia † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	2/57 (3.51%)	2
Anal pruritus † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Aphthous stomatitis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Chapped lips † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Constipation † 1	4/60 (6.67%)	6	2/57 (3.51%)	3	2/57 (3.51%)	2
Diarrhoea † 1	18/60 (30.00%)	39	11/57 (19.30%)	15	15/57 (26.32%)	20
Dyspepsia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Dysphagia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Enteritis † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	1/57 (1.75%)	1
Faecal incontinence † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Flatulence † 1	10/60 (16.67%)	10	5/57 (8.77%)	5	4/57 (7.02%)	4
Frequent bowel movements † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Gastritis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Gastrooesophageal reflux disease † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Gingival bleeding † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Gingivitis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Haematemesis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Ileus † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Lip dry † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Mouth ulceration † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Nausea † 1	10/60 (16.67%)	16	8/57 (14.04%)	16	7/57 (12.28%)	9
Oral pain † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Reflux oesophagitis † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Regurgitation † 1	1/60 (1.67%)	1	2/57 (3.51%)	4	0/57 (0.00%)	0
Stomach discomfort † 1	5/60 (8.33%)	7	4/57 (7.02%)	4	0/57 (0.00%)	0
Tooth disorder † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Tooth impacted † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Tooth loss † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Toothache † 1	1/60 (1.67%)	1	2/57 (3.51%)	2	1/57 (1.75%)	1
Vomiting † 1	28/60 (46.67%)	72	33/57 (57.89%)	88	22/57 (38.60%)	48
General disorders
Adhesion † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Asthenia † 1	4/60 (6.67%)	5	3/57 (5.26%)	4	2/57 (3.51%)	2
Disease progression † 1	5/60 (8.33%)	5	4/57 (7.02%)	4	6/57 (10.53%)	6
Energy increased † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Fatigue † 1	6/60 (10.00%)	6	2/57 (3.51%)	2	2/57 (3.51%)	3
Feeling abnormal † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Feeling hot † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Gait disturbance † 1	4/60 (6.67%)	4	0/57 (0.00%)	0	1/57 (1.75%)	1
Ill-defined disorder † 1	1/60 (1.67%)	4	0/57 (0.00%)	0	0/57 (0.00%)	0
Malaise † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	1/57 (1.75%)	1
Pain † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Pyrexia † 1	8/60 (13.33%)	10	14/57 (24.56%)	21	12/57 (21.05%)	14
Thirst † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Immune system disorders
Drug hypersensitivity † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Hypersensitivity † 1	1/60 (1.67%)	2	0/57 (0.00%)	0	0/57 (0.00%)	0
Seasonal allergy † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	4/57 (7.02%)	4
Infections and infestations
Bronchitis † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	5/57 (8.77%)	5
Catheter site infection † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Croup infectious † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Cystitis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Ear infection † 1	4/60 (6.67%)	5	3/57 (5.26%)	4	4/57 (7.02%)	4
Eczema infected † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Enterobiasis † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Eyelid infection † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Fungal infection † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Gastroenteritis † 1	6/60 (10.00%)	7	10/57 (17.54%)	10	4/57 (7.02%)	7
Gastroenteritis viral † 1	3/60 (5.00%)	3	4/57 (7.02%)	4	3/57 (5.26%)	4
Genital candidiasis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Herpes zoster † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	2
Hordeolum † 1	2/60 (3.33%)	4	0/57 (0.00%)	0	0/57 (0.00%)	0
Influenza † 1	7/60 (11.67%)	9	6/57 (10.53%)	8	8/57 (14.04%)	9
Infusion site infection † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Lice infestation † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Localised infection † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Lower respiratory tract infection † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	1/57 (1.75%)	1
Measles † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Molluscum contagiosum † 1	1/60 (1.67%)	1	2/57 (3.51%)	3	0/57 (0.00%)	0
Nasopharyngitis † 1	10/60 (16.67%)	15	13/57 (22.81%)	22	13/57 (22.81%)	17
Oral candidiasis † 1	1/60 (1.67%)	2	0/57 (0.00%)	0	0/57 (0.00%)	0
Oral herpes † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Otitis externa † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Otitis media † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	4/57 (7.02%)	4
Paronychia † 1	0/60 (0.00%)	0	4/57 (7.02%)	4	1/57 (1.75%)	1
Pharyngitis † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	0/57 (0.00%)	0
Pharyngitis streptococcal † 1	2/60 (3.33%)	3	1/57 (1.75%)	3	3/57 (5.26%)	3
Pharyngotonsillitis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Pneumonia † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Post procedural infection † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Postoperative wound infection † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Respiratory tract infection † 1	1/60 (1.67%)	3	0/57 (0.00%)	0	0/57 (0.00%)	0
Rhinitis † 1	3/60 (5.00%)	4	7/57 (12.28%)	11	2/57 (3.51%)	2
Roseola † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Scarlet fever † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Sinusitis † 1	2/60 (3.33%)	2	1/57 (1.75%)	1	4/57 (7.02%)	7
Skin infection † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Tinea capitis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Tinea infection † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Tinea pedis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	4
Tonsillitis † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	0/57 (0.00%)	0
Upper respiratory tract infection † 1	12/60 (20.00%)	18	9/57 (15.79%)	16	12/57 (21.05%)	17
Urinary tract infection † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Varicella † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	1/57 (1.75%)	1
Viral infection † 1	3/60 (5.00%)	4	3/57 (5.26%)	3	1/57 (1.75%)	1
Viral pharyngitis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Viral upper respiratory tract infection † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	0/57 (0.00%)	0
Injury, poisoning and procedural complications
Accident † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Accidental overdose † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Arthropod bite † 1	1/60 (1.67%)	1	1/57 (1.75%)	2	3/57 (5.26%)	3
Arthropod sting † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Back injury † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Chillblains † 1	0/60 (0.00%)	0	1/57 (1.75%)	2	0/57 (0.00%)	0
Contusion † 1	8/60 (13.33%)	9	8/57 (14.04%)	10	4/57 (7.02%)	4
Excoriation † 1	3/60 (5.00%)	3	0/57 (0.00%)	0	4/57 (7.02%)	6
Eye injury † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Face injury † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Fall † 1	7/60 (11.67%)	14	11/57 (19.30%)	15	7/57 (12.28%)	7
Femur fracture † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	2
Foot fracture † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Hand fracture † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Head injury † 1	2/60 (3.33%)	3	0/57 (0.00%)	0	0/57 (0.00%)	0
Humerus fracture † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Iliotibial band syndrome † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Incision site erythema † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Joint injury † 1	0/60 (0.00%)	0	5/57 (8.77%)	5	1/57 (1.75%)	1
Joint sprain † 1	4/60 (6.67%)	4	4/57 (7.02%)	4	1/57 (1.75%)	1
Laceration † 1	1/60 (1.67%)	2	0/57 (0.00%)	0	0/57 (0.00%)	0
Limb injury † 1	0/60 (0.00%)	0	5/57 (8.77%)	6	1/57 (1.75%)	1
Lower limb fracture † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Lumbar vertebral fracture † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Muscle strain † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Post procedural discomfort † 1	3/60 (5.00%)	3	6/57 (10.53%)	8	0/57 (0.00%)	0
Post procedural haematoma † 1	4/60 (6.67%)	4	3/57 (5.26%)	3	2/57 (3.51%)	2
Post procedural swelling † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Procedural pain † 1	11/60 (18.33%)	17	10/57 (17.54%)	13	10/57 (17.54%)	14
Scratch † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Skin laceration † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	3/57 (5.26%)	3
Spinal compression fracture † 1	0/60 (0.00%)	0	1/57 (1.75%)	2	0/57 (0.00%)	0
Sunburn † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Suture rupture † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Thermal burn † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Tibia fracture † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	2
Tooth fracture † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Tooth injury † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Traumatic haematoma † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Wound † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	2/57 (3.51%)	2
Wound dehiscence † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Wrist fracture † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Investigations
Blood aldosterone increased † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Blood alkaline phosphatase increased † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Blood bicarbonate abnormal † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Blood bicarbonate decreased † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Blood calcium increased † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Blood magnesium increased † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Blood pressure increased † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Immunoglobulins decreased † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Renin increased † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Thyroxine free increased † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Thyroxine increased † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Urine colour abnormal † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	3
Weight decreased † 1	5/60 (8.33%)	5	6/57 (10.53%)	6	1/57 (1.75%)	1
Weight increased † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Metabolism and nutrition disorders
Decreased appetite † 1	5/60 (8.33%)	6	5/57 (8.77%)	6	2/57 (3.51%)	2
Dehydration † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Hyperuricaemia † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Iron deficiency † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Musculoskeletal and connective tissue disorders
Arthralgia † 1	7/60 (11.67%)	9	2/57 (3.51%)	2	2/57 (3.51%)	2
Back pain † 1	6/60 (10.00%)	15	9/57 (15.79%)	9	5/57 (8.77%)	10
Coccydynia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Groin pain † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	3
Joint contracture † 1	3/60 (5.00%)	3	3/57 (5.26%)	3	0/57 (0.00%)	0
Joint swelling † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Lordosis † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	1/57 (1.75%)	1
Muscle contracture † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Muscle spasms † 1	1/60 (1.67%)	1	3/57 (5.26%)	4	5/57 (8.77%)	8
Muscle tightness † 1	1/60 (1.67%)	1	2/57 (3.51%)	2	1/57 (1.75%)	1
Muscular weakness † 1	5/60 (8.33%)	5	3/57 (5.26%)	3	2/57 (3.51%)	2
Musculoskeletal chest pain † 1	1/60 (1.67%)	1	2/57 (3.51%)	4	1/57 (1.75%)	1
Musculoskeletal pain † 1	2/60 (3.33%)	2	1/57 (1.75%)	1	1/57 (1.75%)	1
Myalgia † 1	2/60 (3.33%)	2	3/57 (5.26%)	3	2/57 (3.51%)	2
Neck pain † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Osteoporosis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Pain in extremity † 1	8/60 (13.33%)	16	8/57 (14.04%)	12	6/57 (10.53%)	7
Scoliosis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Tendinous contracture † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Tendon disorder † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	1/57 (1.75%)	1
Trendelenburg's symptom † 1	1/60 (1.67%)	2	1/57 (1.75%)	1	0/57 (0.00%)	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Skin papilloma † 1	1/60 (1.67%)	1	2/57 (3.51%)	3	1/57 (1.75%)	1
Nervous system disorders
Areflexia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Convulsion † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Dizziness † 1	3/60 (5.00%)	3	3/57 (5.26%)	4	4/57 (7.02%)	5
Headache † 1	16/60 (26.67%)	85	23/57 (40.35%)	69	14/57 (24.56%)	25
Hypertonia † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Hypotonia † 1	2/60 (3.33%)	2	1/57 (1.75%)	1	0/57 (0.00%)	0
Lethargy † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Migraine † 1	1/60 (1.67%)	2	2/57 (3.51%)	4	0/57 (0.00%)	0
Psychomotor hyperactivity † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Sinus headache † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Syncope † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Unresponsive to stimuli † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Psychiatric disorders
Abnormal behaviour † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Aggression † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Agitation † 1	1/60 (1.67%)	3	1/57 (1.75%)	1	0/57 (0.00%)	0
Anger † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Anxiety † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	1/57 (1.75%)	2
Attention deficit/hyperactivity disorder † 1	3/60 (5.00%)	3	2/57 (3.51%)	2	0/57 (0.00%)	0
Depression † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Emotional disorder † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Encopresis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Initial insomnia † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Insomnia † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	3
Mood swings † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Obsessive-compulsive disorder † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Oppositional defiant disorder † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Sleep disorder † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Somnambulism † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Stress † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Tic † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	2
Renal and urinary disorders
Dysuria † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	1/57 (1.75%)	1
Enuresis † 1	3/60 (5.00%)	4	4/57 (7.02%)	4	0/57 (0.00%)	0
Incontinence † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Myoglobinuria † 1	1/60 (1.67%)	1	1/57 (1.75%)	3	0/57 (0.00%)	0
Pollakiuria † 1	2/60 (3.33%)	2	1/57 (1.75%)	1	1/57 (1.75%)	1
Pyelocaliectasis † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Renal cyst † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Residual urine † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Strangury † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Ureteric dilatation † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Urinary incontinence † 1	2/60 (3.33%)	2	2/57 (3.51%)	2	2/57 (3.51%)	2
Urine abnormality † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Reproductive system and breast disorders
Balanitis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Genital erythema † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Pruritus genital † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Testicular pain † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Respiratory, thoracic and mediastinal disorders
Asthma † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Bronchial hyperreactivity † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Cough † 1	15/60 (25.00%)	27	9/57 (15.79%)	14	13/57 (22.81%)	19
Dyspnoea † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Epistaxis † 1	3/60 (5.00%)	7	3/57 (5.26%)	6	1/57 (1.75%)	1
Nasal congestion † 1	6/60 (10.00%)	8	5/57 (8.77%)	8	5/57 (8.77%)	9
Nasal discomfort † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Oropharyngeal pain † 1	4/60 (6.67%)	7	6/57 (10.53%)	7	4/57 (7.02%)	7
Productive cough † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Pulmonary congestion † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Rhinitis allergic † 1	0/60 (0.00%)	0	1/57 (1.75%)	3	0/57 (0.00%)	0
Rhinorrhoea † 1	1/60 (1.67%)	1	4/57 (7.02%)	7	6/57 (10.53%)	8
Sinus congestion † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	2
Sneezing † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Throat irritation † 1	1/60 (1.67%)	1	1/57 (1.75%)	2	0/57 (0.00%)	0
Upper respiratory tract congestion † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	2
Wheezing † 1	2/60 (3.33%)	4	0/57 (0.00%)	0	1/57 (1.75%)	1
Skin and subcutaneous tissue disorders
Acne † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Decubitus ulcer † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Dermatitis allergic † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Dermatitis contact † 1	1/60 (1.67%)	1	1/57 (1.75%)	1	0/57 (0.00%)	0
Dry skin † 1	0/60 (0.00%)	0	2/57 (3.51%)	2	3/57 (5.26%)	3
Ecchymosis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Eczema † 1	2/60 (3.33%)	3	1/57 (1.75%)	1	2/57 (3.51%)	2
Erythema † 1	1/60 (1.67%)	1	3/57 (5.26%)	4	0/57 (0.00%)	0
Exfoliative rash † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Heat rash † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Hyperhidrosis † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Hyperkeratosis † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Ingrowing nail † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Keratosis pilaris † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Nail disorder † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Night sweats † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Pruritus † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	2/57 (3.51%)	2
Rash † 1	8/60 (13.33%)	8	6/57 (10.53%)	7	5/57 (8.77%)	6
Rash erythematous † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Rash follicular † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Rash generalised † 1	0/60 (0.00%)	0	0/57 (0.00%)	0	1/57 (1.75%)	1
Rash maculo-papular † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Rash papular † 1	2/60 (3.33%)	2	0/57 (0.00%)	0	1/57 (1.75%)	1
Scar † 1	5/60 (8.33%)	9	4/57 (7.02%)	8	4/57 (7.02%)	7
Skin discolouration † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Skin irritation † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Skin lesion † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Urticaria † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	1/57 (1.75%)	1
Surgical and medical procedures
Endodontic procedure † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Tooth extraction † 1	0/60 (0.00%)	0	1/57 (1.75%)	1	0/57 (0.00%)	0
Vascular disorders
Flushing † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	1/57 (1.75%)	1
Hypertension † 1	0/60 (0.00%)	0	4/57 (7.02%)	6	1/57 (1.75%)	1
Hypotension † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
Pallor † 1	1/60 (1.67%)	1	0/57 (0.00%)	0	0/57 (0.00%)	0
† Indicates events were collected by systematic assessment; 1 Term from vocabulary, MedDRA 9.1

Limitations and Caveats

[Not Specified]

More Information

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

• Name/Title: Patient Advocacy
• Organization: PTC Therapeutics, Inc.
• Phone: 1-866-562-4620
• EMail: medinfo@ptcbio.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Campbell C, Barohn RJ, Bertini E, Chabrol B, Comi GP, Darras BT, Finkel RS, Flanigan KM, Goemans N, Iannaccone ST, Jones KJ, Kirschner J, Mah JK, Mathews KD, McDonald CM, Mercuri E, Nevo Y, Péréon Y, Renfroe JB, Ryan MM, Sampson JB, Schara U, Sejersen T, Selby K, Tulinius M, Vílchez JJ, Voit T, Wei LJ, Wong BL, Elfring G, Souza M, McIntosh J, Trifillis P, Peltz SW, Muntoni F; PTC124-GD-007-DMD Study Group; ACT DMD Study Group; Clinical Evaluator Training Groups. Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy. J Comp Eff Res. 2020 Oct;9(14):973-984. doi: 10.2217/cer-2020-0095. Epub 2020 Aug 27.

• Responsible Party: PTC Therapeutics
• ClinicalTrials.gov Identifier: NCT00592553
• Other Study ID Numbers: PTC124-GD-007-DMD; 2007-005478-29 ( EudraCT Number )
• First Submitted: January 1, 2008
• First Posted: January 14, 2008
• Results First Submitted: March 11, 2020
• Results First Posted: April 7, 2020
• Last Update Posted: April 7, 2020