for people ages 4 years and up (full criteria)
at La Jolla, California and other locations
study started
completion around
Principal Investigator
by Chamindra Laverty, MD



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).

Official Title

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4


Limb-girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2E/R4, LGMD2E/R4, Pediatric, SRP-9003, scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec, Non-ambulatory, Ambulatory, Muscular Dystrophies, Limb-Girdle Muscular Dystrophies, Prednisone, Glucocorticoids, Glucocorticoid


You can join if…

Open to people ages 4 years and up

  • Cohort 1, only ambulatory participants:
    • Able to walk without assistive aid
    • 10-meter walk test (10MWT) <30 seconds
    • NSAD ≥25
  • Cohort 2, only non-ambulatory participants:
    • 10MWT ≥30 seconds or unable to perform
    • PUL 2.0 entry scale score ≥3
  • Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
  • Able to cooperate with muscle testing
  • Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

You CAN'T join if...

  • Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
  • Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
  • Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
  • Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria apply.


  • University of California, San Diego-Altman Clinical and Translational Research Institute accepting new patients
    La Jolla California 92037 United States
  • Nationwide Childrens Hospital accepting new patients
    Columbus Ohio 43215 United States
  • Children's Hospital of The King's Daughter accepting new patients
    Norfolk Virginia 23507 United States
  • The Children's Hospital of Philadelphia accepting new patients
    Philadelphia Pennsylvania 19104 United States

Lead Scientist at UCSD


accepting new patients
Start Date
Completion Date
Sarepta Therapeutics, Inc.
Phase 3 Limb-Girdle Muscular Dystrophy Research Study
Study Type
Expecting 15 study participants
Last Updated