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NCT01826487 · ClinicalTrials.gov registry record · Phase 3
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
A Phase 3 study, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 230
- Enrollment target
NCT01826487 is a Phase 3 study that has completed, run by PTC Therapeutics. The registered enrollment target is 230 participants.
The verdict
NCT01826487, a Phase 3 study, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 230 participants
- Enrollment target
Study Summary
Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.
Interventions
- DRUG Placebo
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 230 participants |
| Start Date | 2013-03-26 |
| Est. Completion | 2015-08-20 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01826487
The ClinicalTrials.gov registry entry for NCT01826487 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 230 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01826487 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01826487 about?
NCT01826487 is a clinical study titled "Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)". Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to...
What is the current status of trial NCT01826487?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 230 participants. The study started on 2013-03-26. Estimated completion is 2015-08-20.
What interventions are being tested in trial NCT01826487?
The interventions under investigation include: Placebo (DRUG), Ataluren (DRUG).
Who is sponsoring clinical trial NCT01826487?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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