Trial · NCT01826487

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

NCT01826487, Phase 3, Completed, PTC Therapeutics; results first posted 2020-08-04

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD). Trials, record NCT01826487. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01826487

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
NCT number: NCT01826487
Status: Completed
Phases: Phase 3
Enrollment: 230 participants, actual
Lead sponsor: PTC Therapeutics
Conditions: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions: Ataluren, Placebo
First posted: 2013-04-08
Last updated: 2020-08-04
Other registries' IDs: eudract:2012-004527-20
Results first posted: 2020-08-04

Source ClinicalTrials.gov

Registered outcomes11

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in 6MWD at Week 48	Baseline, Week 48	The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline and Week 48 6MWD values are each the average of two valid 6MWD values, or a single available value if one was missing.
Secondary	Time to 10 Percent (%) Persistent Worsening in 6MWD	Baseline to Week 48	The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Time to 10% persistent worsening in 6MWD \<300 meters, \>=300 to 400 meters, and \>=400 meters was evaluated. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of the last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening.
Secondary	Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Baseline, Week 48	During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers \[knee-ankle-foot orthoses \] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used. A cumulative change from baseline data has been reported.
Secondary	Change From Baseline in Time to Climb 4 Stairs at Week 48	Baseline, Week 48	During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.
Secondary	Change From Baseline in Time to Descend 4 Stairs at Week 48	Baseline, Week 48	During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported.
Secondary	Percentage of Participants With Treatment-Emergent Adverse Events (AEs)	Baseline up to Week 54	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'.
Other	Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48	Baseline, Week 48	Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.
Other	Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel	Baseline, Week 48	Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 48), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much worse), 2 (slightly worse), 3 (unchanged), 4 (slightly better), or 5 (much better).
Other	Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48	Baseline, Week 48	Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domains were prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.
Other	Ataluren Plasma Concentration	Weeks 8, 16, 24, 32, 40, and 48	Plasma samples for the determination of ataluren concentrations were analyzed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation of 0.5 micrograms/milliliter (mcg/mL).

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in 6MWD at Week 48	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Multiple imputation was applied to impute missing values within the treatment groups.	meters	LEAST_SQUARES_MEAN
SECONDARY	Time to 10 Percent (%) Persistent Worsening in 6MWD	Baseline to Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Multiple imputation was applied to impute missing values within treatment groups. Here, 'Number analyzed 'signifies participants evaluable for specified categories.	days	MEDIAN
SECONDARY	Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Baseline, Week 48	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.	seconds	MEAN
SECONDARY	Change From Baseline in Time to Climb 4 Stairs at Week 48	Baseline, Week 48	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.	seconds	MEAN
SECONDARY	Change From Baseline in Time to Descend 4 Stairs at Week 48	Baseline, Week 48	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.	seconds	MEAN
OTHER_PRE_SPECIFIED	Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48	Baseline, Week 48	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.	units on a scale	MEAN
OTHER_PRE_SPECIFIED	Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel	Baseline, Week 48	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.	Participants	COUNT_OF_PARTICIPANTS
OTHER_PRE_SPECIFIED	Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48	Baseline, Week 48	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.	units on a scale	MEAN
OTHER_PRE_SPECIFIED	Ataluren Plasma Concentration	Weeks 8, 16, 24, 32, 40, and 48	As-treated population included all randomized participants who actually received any study treatment. Here, 'Number analyzed 'signifies participants evaluable at specified timepoint.	mcg/mL	MEAN
OTHER_PRE_SPECIFIED	Study Drug Compliance	Baseline to Week 48	As-treated population included all randomized participants who actually received any study treatment.	percentage of drug	MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group120

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in 6MWD at Week 48	Placebo		-60.67	Standard Error 9.323	meters
Change From Baseline in 6MWD at Week 48	Ataluren		-47.69	Standard Error 9.247	meters
Time to 10 Percent (%) Persistent Worsening in 6MWD	Placebo	<300 meters	56	95% Confidence Interval 1.0 to 111.0	days
Time to 10 Percent (%) Persistent Worsening in 6MWD	Ataluren	<300 meters	164	95% Confidence Interval 1.0 to 225.0	days
Time to 10 Percent (%) Persistent Worsening in 6MWD	Placebo	>=300 to <400 meters	280	95% Confidence Interval 169.0 to NA	days
Time to 10 Percent (%) Persistent Worsening in 6MWD	Ataluren	>=300 to <400 meters	NA	95% Confidence Interval 280.0 to NA	days
Time to 10 Percent (%) Persistent Worsening in 6MWD	Placebo	>=400 meters	NA	95% Confidence Interval NA to NA	days
Time to 10 Percent (%) Persistent Worsening in 6MWD	Ataluren	>=400 meters	NA	95% Confidence Interval NA to NA	days
Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Placebo		3.47	Standard Deviation 6.393	seconds
Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Ataluren		2.27	Standard Deviation 5.216	seconds

1 to 10 of 120Source ClinicalTrials.gov

See all 120 results by group

Participant flow10

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	115
Overall Study	STARTED	Ataluren	115
Overall Study	As-treated Population	Placebo	115
Overall Study	As-treated Population	Ataluren	115
Overall Study	ITT Population	Placebo	114
Overall Study	ITT Population	Ataluren	114
Overall Study	COMPLETED	Placebo	111
Overall Study	COMPLETED	Ataluren	110
Overall Study	NOT COMPLETED	Placebo	4
Overall Study	NOT COMPLETED	Ataluren	5

Source ClinicalTrials.gov

Adverse events518

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Neutropenia	Blood and lymphatic system disorders	Placebo	0	115
other	Neutropenia	Blood and lymphatic system disorders	Ataluren	1	115
other	Splenomegaly	Blood and lymphatic system disorders	Placebo	0	115
other	Splenomegaly	Blood and lymphatic system disorders	Ataluren	1	115
other	Leukocytosis	Blood and lymphatic system disorders	Placebo	1	115
other	Leukocytosis	Blood and lymphatic system disorders	Ataluren	0	115
other	Lymphadenopathy	Blood and lymphatic system disorders	Placebo	1	115
other	Lymphadenopathy	Blood and lymphatic system disorders	Ataluren	0	115
other	Right ventricular hypertrophy	Cardiac disorders	Placebo	0	115
other	Right ventricular hypertrophy	Cardiac disorders	Ataluren	2	115

1 to 10 of 518Source ClinicalTrials.gov

See all 518 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo
Total, all-cause mortality	Ataluren
Total, other adverse events	Placebo	99	115
Total, other adverse events	Ataluren	103	115
Total, serious adverse events	Placebo	4	115
Total, serious adverse events	Ataluren	4	115

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
28728956	DERIVED	PubMed
32851872	DERIVED	PubMed
34383312	DERIVED	PubMed
34693725	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Ataluren vs Placebo: serious adverse events

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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