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
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 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
University Hospital Leuven (UZ Leuven)
Leuven, Vlaams Brabant 3000
BelgiumSite Not Available
University Hospital Leuven (UZ Leuven)
Leuven 2792482, Vlaams Brabant 3000
BelgiumSite Not Available
NMRC Gent (UZ Gent)
Gent, 9000
BelgiumSite Not Available
NMRC Gent (UZ Gent)
Ghent 2797656, 9000
BelgiumSite Not Available
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen, Nordrhein Westfalen 45147
GermanySite Not Available
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen 2928810, North Rhine-Westphalia 2861876 45147
GermanySite Not Available
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan 6951411, 20122
ItalySite Not Available
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milano, 20122
ItalySite Not Available
Hospital Sant Joan de Deu
Barcelona, 8950
SpainSite Not Available
Hospital Sant Joan de Deu
Barcelona 3128760, 8950
SpainSite Not Available
Newcastle University
Newcastle upon Tyne, NE1 3BZ
United KingdomSite Not Available
Newcastle University
Newcastle upon Tyne 2641673, 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
University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla 5363943, California 5332921 92037
United StatesSite Not Available
Nationwide Childrens Hospital
Columbus, Ohio 43205
United StatesSite Not Available
Nationwide Childrens Hospital
Columbus 4509177, Ohio 5165418 43205
United StatesSite Not Available
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
The Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesSite Not Available
Children's Hospital of The King's Daughter
Norfolk, Virginia 23507
United StatesSite Not Available
Children's Hospital of The King's Daughter
Norfolk 4776222, Virginia 6254928 23507
United StatesSite Not Available

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