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NCT06246513 · ClinicalTrials.gov registry record · Phase 3
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
A Phase 3 study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 17
- Enrollment target
- 10
- Study locations
NCT06246513: Active Phase 3 study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
NCT06246513 is a Phase 3 study of Limb Girdle Muscular Dystrophy that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 17 participants, below the 237-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (93% lower). The trial reports 10 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06246513, a Phase 3 study of Limb Girdle Muscular Dystrophy, is active but no longer recruiting, sponsored by Sarepta Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 17 participants
- Enrollment target
- 10
- Study locations
Study Summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Conditions Studied
Interventions
- DRUG Glucocorticoid
- BIOLOGICAL SRP-9003
Study Locations (10)
Other
- NMRC Gent (UZ Gent) - Ghent
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico - Milan
- Hospital Sant Joan de Deu - Barcelona
- Newcastle University - Newcastle upon Tyne
California
- University of California, San Diego-Altman Clinical and Translational Research Institute - La Jolla
Ohio
- Nationwide Childrens Hospital - Columbus
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Virginia
- Children's Hospital of The King's Daughter - Norfolk
Vlaams Brabant
- University Hospital Leuven (UZ Leuven) - Leuven
North Rhine-Westphalia
- Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum - Essen
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2024-01-15 |
| Est. Completion | 2029-11-30 |
| Phase | Phase 3 |
What the registry record for NCT06246513 still lists
NCT06246513 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 237-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (93% lower).
The record links to 1 condition, with Limb Girdle Muscular Dystrophy appearing as the primary indexed condition, and to 2 interventions - of which Glucocorticoid is the first listed.
NCT06246513 lists 10 locations in 7 states (Other, California, Ohio).
Frequently Asked Questions
What is clinical trial NCT06246513 about?
NCT06246513 is a clinical study titled "A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4". This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory parti...
What is the current status of trial NCT06246513?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 17 participants. The study started on 2024-01-15. Estimated completion is 2029-11-30.
What conditions does trial NCT06246513 study?
This clinical trial studies the following conditions: Limb Girdle Muscular Dystrophy.
What interventions are being tested in trial NCT06246513?
The interventions under investigation include: Glucocorticoid (DRUG), SRP-9003 (BIOLOGICAL).
Who is sponsoring clinical trial NCT06246513?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06246513 being conducted?
This trial has 10 study locations across California, Ohio, Pennsylvania, Virginia, Vlaams Brabant. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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