A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

PHASE1Active, not recruitingINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

December 19, 2022

Primary Completion Date

August 28, 2028

Study Completion Date

August 28, 2028

Conditions

Limb Girdle Muscular Dystrophy

Interventions

GENETIC

SRP-9003

Single IV infusion of SRP-9003

Trial Locations (2)

St. Jude Children's Research Hospital, Memphis

Nationwide Children's Hospital, Columbus

All Listed Sponsors

lead

Sarepta Therapeutics, Inc.

INDUSTRY