Summary

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

Description

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).

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

Keywords

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

Eligibility

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.

Locations

  • 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

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sarepta Therapeutics, Inc.
ID
NCT06246513
Phase
Phase 3 Limb-Girdle Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 15 study participants
Last Updated