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NCT03375164 · ClinicalTrials.gov registry record · Phase 1
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
- 1
- Study location
NCT03375164: Completed Phase 1 study of Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics.
NCT03375164 is a Phase 1 study of Duchenne Muscular Dystrophy that has completed, run by Sarepta Therapeutics. The registered enrollment target is 4 participants, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03375164, a Phase 1 study of Duchenne Muscular Dystrophy, has completed, sponsored by Sarepta Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
- 1
- Study location
Study Summary
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.
Primary Outcome
An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events m
Conditions Studied
Interventions
- GENETIC delandistrogene moxeparvovec
Study Locations (1)
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2018-01-04 |
| Est. Completion | 2023-04-25 |
| Phase | Phase 1 |
What the finished NCT03375164 record still lists
NCT03375164 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 412-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which delandistrogene moxeparvovec is the first listed.
NCT03375164 reports a single indexed study location in Ohio.
Frequently Asked Questions
What is clinical trial NCT03375164 about?
NCT03375164 is a clinical study titled "A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)". This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3...
What is the current status of trial NCT03375164?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2018-01-04. Estimated completion is 2023-04-25.
What conditions does trial NCT03375164 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT03375164?
The interventions under investigation include: delandistrogene moxeparvovec (GENETIC).
Who is sponsoring clinical trial NCT03375164?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03375164 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Duchenne Muscular Dystrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03375164's enrollment target sits among peer trials
4 48th of 48 the lowest of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy 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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