Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
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Phase
Phase 3
Status
Completed
Study type
Interventional
Design
Na · None · Treatment
Enrollment
270estimated
Sites
34
Countries
Canada, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2010-11-30 | actual | When the study began enrolling |
| Primary completion | 2026-02-10 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-02-10 | actual | The whole study's end, after follow-up |
| First posted | 2010-11-24 | estimated | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-09 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Duchenne Muscular Dystrophy
Intervention
- Drug: Ataluren — also filed as PTC124, Translarna
Primary outcomes
what the primary-completion date above is the date OFNumber of Participants With Adverse Events
measured Baseline up to end of study (up to approximately 8 years)
Number of Participants With Laboratory Parameters Abnormalities
measured Baseline up to end of study (up to approximately 8 years)
Number of Participants With Abnormal Physical Findings
measured Baseline up to end of study (up to approximately 8 years)
Publications
- PMID 17389552 — reports results
- PMID 17450125 — reports results
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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