NCT01247207Phase 3Completed

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

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 completion2026-02-10Actual

Enrollment · participants270Estimated

Source updated2026-03-09Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Posted 2026-03-09 · 3 fields changed

Completion date type

Recorded edit

− Previous value

Estimated

+ Updated value

Actual

Primary completion date

Recorded edit

− Previous value

2025-04-30

+ Updated value

2026-02-10

Recruitment status

Recorded edit

− Previous value

Enrolling by invitation

+ Updated value

Completed

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Study details

Conditions studied

Duchenne Muscular Dystrophy

Treatments or interventions studied

AtalurenDrugAlso known as PTC124, Translarna

Primary outcomes · main study measures

Read the study measures

measure: Number of Participants With Adverse Events; timeFrame: Baseline up to end of study (up to approximately 8 years); measure: Number of Participants With Laboratory Parameters Abnormalities; description: Laboratory parameters include hematological, serum biochemistry, adrenal laboratories, and urine data.; timeFrame: Baseline up to end of study (up to approximately 8 years); measure: Number of Participants With Abnormal Physical Findings; description: Physical findings include weight, physical examination data, systolic and diastolic blood pressure, radial pulse rate, and body temperature.; timeFrame: Baseline up to end of study (up to approximately 8 years)

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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