clinical trial · NCT06246513
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
Limb-girdle Muscular DystrophySRP-9003Glucocorticoid
brief 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).
started
Jan 15, 2024
primary completion
Mar 4, 2025
completion
Nov 30, 2029
last updated
Mar 2, 2026
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol