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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

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NCT06246513

Sponsored by Sarepta Therapeutics, Inc. (industry) · SRPT — their whole pipeline →.

Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
17actual
Sites
10
Countries
Belgium, Germany, Italy +3

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
DateFiledBasisWhat it is
Start2024-01-15actualWhen the study began enrolling
Primary completion2025-03-04actualThe date the last participant is measured for the primary outcome — the readout window
Study completion2029-11-30estimatedThe whole study's end, after follow-up
First posted2024-02-07actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-03-02actualThe 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

Interventions

  • Biological: SRP-9003 — also filed as scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec
  • Drug: Glucocorticoid — also filed as Prednisone or equivalent

Primary outcome

what the primary-completion date above is the date OF
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
measured Baseline, Day 60

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