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NCT05876780 · ClinicalTrials.gov registry record · Phase 1
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
A Phase 1 study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 2
- Study locations
NCT05876780: Active Phase 1 study of Limb Girdle Muscular Dystrophy, sponsored by Sarepta Therapeutics.
NCT05876780 is a Phase 1 study of Limb Girdle Muscular Dystrophy that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 6 participants, below the 239-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (97% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05876780, a Phase 1 study of Limb Girdle Muscular Dystrophy, is active but no longer recruiting, sponsored by Sarepta Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 2
- Study locations
Study Summary
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
Conditions Studied
Interventions
- GENETIC SRP-9003
Study Locations (2)
Ohio
- Nationwide Children's Hospital - Columbus
Tennessee
- St. Jude Children's Research Hospital - Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2022-12-19 |
| Est. Completion | 2028-08-28 |
| Phase | Phase 1 |
What the registry record for NCT05876780 still lists
NCT05876780 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 239-participant average among 6 other Limb Girdle Muscular Dystrophy trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Limb Girdle Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention - of which SRP-9003 is the first listed.
NCT05876780 reports a single indexed study location in Ohio, Tennessee.
Frequently Asked Questions
What is clinical trial NCT05876780 about?
NCT05876780 is a clinical study titled "A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)". The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
What is the current status of trial NCT05876780?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2022-12-19. Estimated completion is 2028-08-28.
What conditions does trial NCT05876780 study?
This clinical trial studies the following conditions: Limb Girdle Muscular Dystrophy.
What interventions are being tested in trial NCT05876780?
The interventions under investigation include: SRP-9003 (GENETIC).
Who is sponsoring clinical trial NCT05876780?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05876780 being conducted?
This trial has 2 study locations across Ohio, Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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