NCT03179631Phase 3Completed

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2022-03-05Actual

Enrollment · participants360Actual

Source updated2026-03-10Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-01-26 · 1 field changed

Main study measure wording changed

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− Before

Measure: ~~Slope~~ of Change in 6-Minute Walk Distance (6MWD) ~~Over~~ 72 ~~Weeks~~

+ After

Measure: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population Measure: DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population Measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population Measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population

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Study details

Conditions studied

Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Disease; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn

Treatments or interventions studied

AtalurenDrugAlso known as PTC124
PLACEBODrugAlso known as Matching Placebo

Primary outcomes · main study measures

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measure: DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population; description: The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. 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 was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. Least square (LS) mean and standard error (SE) was calculated using the mixed-model repeated measures (MMRM).; timeFrame: Baseline, Week 72; measure: DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population; description: The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. 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 was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.; timeFrame: Baseline, Week 72; measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population; description: The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. 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 was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.; timeFrame: Baseline, Week 72; measure: DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population; description: The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures. 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 was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM.; timeFrame: Baseline, Week 72

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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