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)
← catalyst calendarNCT05876780 · readout in 739 d
Sponsored by Sarepta Therapeutics, Inc. (industry) · SRPT — their whole pipeline →.
Phase
Phase 1
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
6actual
Sites
2
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-12-19 | actual | When the study began enrolling |
| Primary completion | 2028-08-28 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2028-08-28 | estimated | The whole study's end, after follow-up |
| First posted | 2023-05-25 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-02-19 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Limb Girdle Muscular Dystrophy
Intervention
- Genetic: SRP-9003 — also filed as scAAVrh74.MHCK7.hSGCB
Primary outcomes
what the primary-completion date above is the date OFNumber of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
measured Baseline up to Month 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60
measured Baseline, Day 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60
measured Baseline, Day 60
Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60
measured Baseline, Day 60
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