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Clinical Trial 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).


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT06246513
Study type Interventional
Source Sarepta Therapeutics, Inc.
Contact Sarepta Therapeutics Inc. For Clinical Trial Information, Select
Phone 1-888-SAREPTA (1-888-727-3782)
Email SareptAlly@sarepta.com
Status Recruiting
Phase Phase 3
Start date January 15, 2024
Completion date November 30, 2029

See also
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Completed NCT00873782 - Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy Phase 1
Completed NCT00457912 - Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy N/A
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Completed NCT04054375 - Weekly Steroids in Muscular Dystrophy Phase 2
Recruiting NCT00390104 - Molecular Analysis of Patients With Neuromuscular Disease
Completed NCT00104078 - Study Evaluating MYO-029 in Adult Muscular Dystrophy Phase 1/Phase 2
Recruiting NCT04475926 - A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
Completed NCT02759302 - MRI on Persons With Mutations in POMT2 Gene (LGMD2N) N/A