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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

NCT06246513 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2026-03-02

No results posted yet for this 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

  • Limb-girdle Muscular Dystrophy

Interventions

BIOLOGICAL

SRP-9003

Solution for single IV infusion

DRUG

Glucocorticoid

Oral tablet (prophylactic)

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-15
Primary Completion
2025-03-04
Completion
2029-11-30
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Germany
  • Italy
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06246513 on ClinicalTrials.gov