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 (EMERGENE)

Limb-girdle Muscular Dystrophy Clinical Trial

Official title:
A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

Status Recruiting

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

Clinical Trial Details

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

View Details

See also
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Completed	`NCT00104078` - Study Evaluating MYO-029 in Adult Muscular Dystrophy	Phase 1/Phase 2
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