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NCT01009294 · ClinicalTrials.gov registry record · Phase 2
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
A Phase 2 study, sponsored by PTC Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 6
- Enrollment target
NCT01009294: Clinical Trial Phase 2 study, sponsored by PTC Therapeutics.
NCT01009294 is a Phase 2 study that was terminated before completion, run by PTC Therapeutics. The registered enrollment target is 6 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01009294, a Phase 2 study, was terminated before completion, sponsored by PTC Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 6 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 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.
Primary Outcome
A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dy
Interventions
- DRUG Ataluren
- DRUG Chronic Corticosteroid Therapy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2010-01-13 |
| Est. Completion | 2010-03-23 |
| Phase | Phase 2 |
Why NCT01009294 stopped before completion
NCT01009294 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 2 interventions - of which Ataluren is the first listed.
NCT01009294 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01009294 about?
NCT01009294 is a clinical study titled "Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/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 NCT01009294?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 6 participants. The study started on 2010-01-13. Estimated completion is 2010-03-23.
What interventions are being tested in trial NCT01009294?
The interventions under investigation include: Ataluren (DRUG), Chronic Corticosteroid Therapy (DRUG).
Who is sponsoring clinical trial NCT01009294?
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 NCT01009294's enrollment target sits among peer trials
6 1996th of 2000 higher than 1 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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