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NCT00847379 · ClinicalTrials.gov registry record · Phase 2
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 173
- Enrollment target
NCT00847379: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT00847379 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 173 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (30% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00847379, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 173 participants
- Enrollment target
Study Summary
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that 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 during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.
Primary Outcome
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation
Interventions
- DRUG Ataluren
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 173 participants |
| Start Date | 2009-01-31 |
| Est. Completion | 2010-05-24 |
| Phase | Phase 2 |
Why NCT00847379 stopped before completion
NCT00847379 is an interventional study that assigns participants to a tested intervention. The registered 173 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (30% higher).
The record links to 0 conditions, and to 1 intervention - of which Ataluren is the first listed.
NCT00847379 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00847379 about?
NCT00847379 is a clinical study titled "Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)". Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that 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 los...
What is the current status of trial NCT00847379?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 173 participants. The study started on 2009-01-31. Estimated completion is 2010-05-24.
What interventions are being tested in trial NCT00847379?
The interventions under investigation include: Ataluren (DRUG).
Who is sponsoring clinical trial NCT00847379?
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 NCT00847379's enrollment target sits among peer trials
173 397th of 2000 higher than 1,604 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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