Phase
Condition
Muscular Dystrophy
Treatment
Glucocorticoid
SRP-9003
Clinical Study ID
Ages > 4 All Genders
Study Summary
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
Connect with a study center
Newcastle University
Newcastle upon Tyne, NE1 3BZ
United KingdomSite Not Available
University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla, California 92037
United StatesSite Not Available
Nationwide Childrens Hospital
Columbus, Ohio 43215
United StatesSite Not Available
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
Children's Hospital of The King's Daughter
Norfolk, Virginia 23507
United StatesSite Not Available
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