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drugset / Trial / NCT06246513

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 ↗

Phase 3 Active not recruiting 17 enrolled Sarepta Therapeutics, Inc.
NaSingle-groupOpen-labelTreatment

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).

Timeline

Start
2024-01-15
Primary completion
2025-03-04
Completion
2029-11-30

Drugs

EvaluationDrugModalityDoseRoute
Subject BIDRIDISTROGENE XEBOPARVOVEC Gene therapy (AAV / viral vector) — Intravenous
Background Prednisone Other / unclassified — Oral

Indications

limb-girdle muscular dystrophy