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NCT06246513Phase 3ACTIVE NOT RECRUITING

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

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 / Drugs
SRP-9003Glucocorticoid
View on ClinicalTrials.gov ↗
Sponsor
SRPT
Sarepta Therapeutics
Gene Therapy
C54/100
Study Details
Enrollment17 participants
Primary Completion—
Lead SponsorSarepta Therapeutics, Inc.
Data Retrieved2026-04-11 02:55:56.327574+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 17 participants
Sponsor Catalyst Profile

Sarepta Therapeutics (SRPT) is the sponsoring company for this trial. BiotechSign currently grades this company C (54/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

SRPT Full Pipeline →All Companies →
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