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
Running, not enrolling · Phase 3
Conditions studied: Limb-girdle Muscular Dystrophy
In brief
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).
Key facts
- Study ID
- NCT06246513
- Run by
- Sarepta Therapeutics, Inc.
- People needed
- 17
- Starts
- 2024-01-15
- Expected to finish
- 2029-11-30
- Last updated by the study team
- 2026-03-02
Who can join
Age: 4 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Cohort 1, only ambulatory participants:
- Able to walk without assistive aid
- 10MWR <30 seconds
- NSAD ≥25
- Cohort 2, only non-ambulatory participants:
- 10MWR ≥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 may not qualify 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.
Where it is running
- University of California, San Diego-Altman Clinical and Translational Research Institute — La Jolla, California, United States
- Nationwide Childrens Hospital — Columbus, Ohio, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Children's Hospital of The King's Daughter — Norfolk, Virginia, United States
- University Hospital Leuven (UZ Leuven) — Leuven, Vlaams Brabant, Belgium
- NMRC Gent (UZ Gent) — Ghent, Belgium
- Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum — Essen, North Rhine-Westphalia, Germany
- Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico — Milan, Italy
- Hospital Sant Joan de Deu — Barcelona, Spain
- Newcastle University — Newcastle upon Tyne, United Kingdom
Full record on ClinicalTrials.gov
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