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)

Sarepta Therapeutics

Status and phase

Active, not recruiting

Phase 3

Conditions

Limb-girdle Muscular Dystrophy

Treatments

Biological: SRP-9003

Drug: Glucocorticoid

Study type

Interventional

Funder types

Industry

Identifiers

NCT06246513

2022-503112-17-00 (Other Identifier)

SRP-9003-301

Details and patient eligibility

About

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).

Enrollment

17 patients

Sex

All

Ages

4+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Cohort 1, only ambulatory participants:
- Able to walk without assistive aid
- 10-meter walk test (10MWT) <30 seconds
- NSAD ≥25
Cohort 2, only non-ambulatory participants:
- 10MWT ≥30 seconds or unable to perform
- PUL 2.0 entry scale score ≥3
Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
Able to cooperate with muscle testing
Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

Exclusion criteria

Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria apply.