Gene therapy trial aims to restore muscle protein in rare muscular dystrophy
NCT ID NCT06246513
Summary
This Phase 3 study is testing a single-dose gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E/R4. The therapy aims to deliver a working copy of the beta-sarcoglycan gene to help muscles produce a crucial protein they lack. The study will measure if the treatment increases this protein in muscle tissue and track changes in muscle strength and mobility over 5 years in both walking and non-walking participants.
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Contacts and locations
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Locations
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Children's Hospital of The King's Daughter
Norfolk, Virginia, 23507, United States
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Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
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Hospital Sant Joan de Deu
Barcelona, 8950, Spain
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NMRC Gent (UZ Gent)
Ghent, 9000, Belgium
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Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
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Newcastle University
Newcastle upon Tyne, NE1 3BZ, United Kingdom
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen, North Rhine-Westphalia, 45147, Germany
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University Hospital Leuven (UZ Leuven)
Leuven, Vlaams Brabant, 3000, Belgium
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University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
Conditions
Explore the condition pages connected to this study.