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

Last updated: April 4, 2025
Sponsor: Sarepta Therapeutics, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Muscular Dystrophy

Treatment

Glucocorticoid

SRP-9003

Clinical Study ID

NCT06246513
SRP-9003-301
2022-503112-17-00
  • Ages > 4
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • 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 likelypathogenic β-SG DNA gene mutations

  • Able to cooperate with muscle testing

  • Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibodytiters <1:400 (that is, not elevated) as determined by AAVrh74 antibodyenzyme-linked immunosorbent assay.

Exclusion

Exclusion Criteria:

  • Left ventricular ejection fraction < 40% or clinical signs and/or symptoms ofcardiomyopathy

  • Forced vital capacity ≤40% of predicted value and/or requirement for nocturnalventilation

  • Diagnosis of (or ongoing treatment for) an autoimmune disease and on activeimmunosuppressant treatment

  • Presence of any other clinically significant illness or medical condition (otherthan LGMD2E/R4)

Other inclusion/exclusion criteria apply.

Study Design

Total Participants: 17
Treatment Group(s): 2
Primary Treatment: Glucocorticoid
Phase: 3
Study Start date:
January 15, 2024
Estimated Completion Date:
November 30, 2029

Connect with a study center

  • Newcastle University

    Newcastle upon Tyne, NE1 3BZ
    United Kingdom

    Site Not Available

  • University of California, San Diego-Altman Clinical and Translational Research Institute

    La Jolla, California 92037
    United States

    Site Not Available

  • Nationwide Childrens Hospital

    Columbus, Ohio 43215
    United States

    Site Not Available

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Children's Hospital of The King's Daughter

    Norfolk, Virginia 23507
    United States

    Site Not Available

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