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NCT04004065 · ClinicalTrials.gov registry record · Phase 2
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
A Phase 2 study, sponsored by Sarepta Therapeutics.
- Terminated
- Registry status
- Phase 2
- Development phase
- 62
- Enrollment target
NCT04004065: Clinical Trial Phase 2 study, sponsored by Sarepta Therapeutics.
NCT04004065 is a Phase 2 study that was terminated before completion, run by Sarepta Therapeutics. The registered enrollment target is 62 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04004065, a Phase 2 study, was terminated before completion, sponsored by Sarepta Therapeutics.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 62 participants
- Enrollment target
Study Summary
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose \[MAD\]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
Interventions
- DRUG Vesleteplirsen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 62 participants |
| Start Date | 2019-06-26 |
| Est. Completion | 2025-02-07 |
| Phase | Phase 2 |
Why NCT04004065 stopped before completion
NCT04004065 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 62 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (53% lower).
The record links to 0 conditions, and to 1 intervention - of which Vesleteplirsen is the first listed.
NCT04004065 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04004065 about?
NCT04004065 is a clinical study titled "Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment". This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose \[MAD\]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses s...
What is the current status of trial NCT04004065?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 62 participants. The study started on 2019-06-26. Estimated completion is 2025-02-07.
What interventions are being tested in trial NCT04004065?
The interventions under investigation include: Vesleteplirsen (DRUG).
Who is sponsoring clinical trial NCT04004065?
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 NCT04004065's enrollment target sits among peer trials
62 1016th of 2000 higher than 975 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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