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NCT01247207 · ClinicalTrials.gov registry record · Phase 3
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
A Phase 3 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 270
- Enrollment target
NCT01247207: Completed Phase 3 study, sponsored by PTC Therapeutics.
NCT01247207 is a Phase 3 study that has completed, run by PTC Therapeutics. The registered enrollment target is 270 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (65% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01247207, a Phase 3 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 270 participants
- Enrollment target
Study Summary
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 270 participants |
| Start Date | 2010-11-30 |
| Est. Completion | 2026-02-10 |
| Phase | Phase 3 |
What the finished NCT01247207 record still lists
NCT01247207 is an interventional study that assigns participants to a tested intervention. The registered 270 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (65% lower).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT01247207 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01247207 about?
NCT01247207 is a clinical study titled "Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)". The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participa...
What is the current status of trial NCT01247207?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 270 participants. The study started on 2010-11-30. Estimated completion is 2026-02-10.
What interventions are being tested in trial NCT01247207?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT01247207?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01247207's enrollment target sits among peer trials
270 1217th of 2000 higher than 781 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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