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)

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Description

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).

Study Overview

Start Date
January 15, 2024
Completion Date
November 30, 2029
Enrollment
15
Date Posted
February 7, 2024
Accepts Healthy Volunteers?
No
Gender
All

Locations

Full Address
Children's Hospital of The King's Daughter
Norfolk, Virginia 23507, United States

Eligibility

Minimum Age (years)
4
Eligibility Criteria
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.

Study Contact Info

Study Contact Name
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Study Contact Email
Study Contact Phone

Contact Listings Owner Form

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Other Details

FDA Regulated Drug?
Yes
FDA Regulated Device?
No
NCTid (if applicable)
NCT06246513