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NCT02710500 · ClinicalTrials.gov registry record · Phase 1
rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies
A Phase 1 study, sponsored by Sarepta Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 2
- Enrollment target
NCT02710500: Completed Phase 1 study, sponsored by Sarepta Therapeutics.
NCT02710500 is a Phase 1 study that has completed, run by Sarepta Therapeutics. The registered enrollment target is 2 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02710500, a Phase 1 study, has completed, sponsored by Sarepta Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 2 participants
- Enrollment target
Study Summary
The proposed clinical trial is a double-blind, randomized controlled study with direct intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum brevis muscle (EDB). Two cohorts of subjects with dysferlin deficiency, each with proven mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each cohort.
Primary Outcome
Defined as the occurrence of any one Grade III or higher, unanticipated, treatment-related toxicity
Interventions
- DRUG rAAVrh74.MHCK7.DYSF.DV
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2016-03 |
| Est. Completion | 2019-07 |
| Phase | Phase 1 |
What the finished NCT02710500 record still lists
NCT02710500 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 2 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAVrh74.MHCK7.DYSF.DV is the first listed.
NCT02710500 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02710500 about?
NCT02710500 is a clinical study titled "rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies". The proposed clinical trial is a double-blind, randomized controlled study with direct intramuscular injection of rAAVrh.74.MHCK7.DYSF.DV gene vector to the extensor digitorum brevis muscle (EDB). Two cohorts of subjects with dysferlin deficiency, each with proven mutations will undergo gene transfe...
What is the current status of trial NCT02710500?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 2 participants. The study started on 2016-03. Estimated completion is 2019-07.
What interventions are being tested in trial NCT02710500?
The interventions under investigation include: rAAVrh74.MHCK7.DYSF.DV (DRUG).
Who is sponsoring clinical trial NCT02710500?
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 NCT02710500's enrollment target sits among peer trials
2 2000th of 2000 the lowest of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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