Overview

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

Status:
ACTIVE_NOT_RECRUITING

Trial end date:
2029-11-30

Target enrollment:

Participant gender:

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).

Phase:

PHASE3

Details

Lead Sponsor:

Sarepta Therapeutics, Inc.

Treatments:

Glucocorticoids
Prednisone