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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

Study acronym: EMERGENE
StatusActive, Not Recruiting
PhasePhase 3
Started2024-01-15
View on ClinicalTrials.gov ↗

Amendment history

2026-02-27
Study Status, Outcome Measures, Eligibility v7
Eligibility criteria-21 characters
Inclusion Criteria: Cohort 1, only ambulatory participants: Able to walk without assistive aid 10-meter walk test (10MWT)10MWR <30 seconds NSAD ≥25 Cohort 2, only non-ambulatory participants: 10MWT10MWR ≥30 seconds or unable to perform PUL 2.0 entry scale score [...]
Secondary endpoints13 entries, revised
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western BlotAssay Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for DysferlinopathyLimb-girdle Muscular Dystrophies (NSAD) Total Score Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
2025-07-09
Study Status, Contacts/Locations v6
Study sites5→10
2025-04-04
Study Status v5
Re-verified, no change to tracked fields
2025-03-02
Study Status v4
Primary completion date2025-03-31→2025-03-04
2025-02-28
Recruiting→Active, Not Recruiting Study Status, Study Design, Contacts/Locations v3
Trial statusRecruiting→Active, Not Recruiting
Primary completion date2025-01-31→2025-03-31
Enrollment targetestimated 15→confirmed 17
Study sitesdetails revised at 5 of 5 sites
Show 2 earlier versions
2024-11-20
Study Identification, Study Status, Outcome Measures, Contacts/Locations v2
Study sites4→5
Secondary endpoints12 to 13 entries
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Blot Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI) Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF) Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 [...] [...]
Primary endpoints1 entry, revised
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose [...]
Study title-11 characters
[...] Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
2024-05-24
Study Status, Outcome Measures, Contacts/Locations v1
Study sites1→4
Secondary endpoints12 entries, reordered
2024-01-30
Original filing
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).
Trial Details
NCT Number NCT06246513
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Limb-girdle Muscular Dystrophy
Enrollment 17 participants
Start Date 2024-01-15
Primary Completion 2025-03-04 (estimated)
Study Completion 2029-11-30 (estimated)
Updated on ClinicalTrials.gov 2026-03-02