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NCT01396239 · ClinicalTrials.gov registry record · Phase 2
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
A Phase 2 study, sponsored by Sarepta Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 12
- Enrollment target
NCT01396239: Completed Phase 2 study, sponsored by Sarepta Therapeutics.
NCT01396239 is a Phase 2 study that has completed, run by Sarepta Therapeutics. The registered enrollment target is 12 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01396239, a Phase 2 study, has completed, sponsored by Sarepta Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 12 participants
- Enrollment target
Study Summary
This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy (DMD).
Primary Outcome
The primary efficacy endpoint will be based on the pre-treatment and post-treatment change in the number (%) of dystrophin positive fibers as measured in the muscle biopsy tissue on immunohistochemistry (IHC).
Interventions
- OTHER Placebo
- DRUG AVI-4658 (Eteplirsen)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2011-07 |
| Est. Completion | 2012-06 |
| Phase | Phase 2 |
What the finished NCT01396239 record still lists
NCT01396239 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT01396239 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01396239 about?
NCT01396239 is a clinical study titled "Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients". This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy (DMD).
What is the current status of trial NCT01396239?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2011-07. Estimated completion is 2012-06.
What interventions are being tested in trial NCT01396239?
The interventions under investigation include: Placebo (OTHER), AVI-4658 (Eteplirsen) (DRUG).
Who is sponsoring clinical trial NCT01396239?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01396239's enrollment target sits among peer trials
12 1948th of 2000 higher than 34 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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