Summary

Eligibility
for people ages 4 years and up (full criteria)
Location
at UCSD
Dates
study started
study ends around

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, Limb-Girdle Muscular Dystrophies, Glucocorticoids, Prednisone, 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
    La Jolla 5363943 California 5332921 92037 United States
  • Nationwide Childrens Hospital
    Columbus 4509177 Ohio 5165418 43205 United States

Details

Status
in progress, not 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
About 17 people participating
Last Updated