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

Phase 3Active Not RecruitingNCT06246513

Sarepta Therapeutics, Inc.17 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Limb-girdle Muscular Dystrophy

Interventions

SRP-9003BIOLOGICAL

Solution for single IV infusion

GlucocorticoidDRUG

Oral tablet (prophylactic)

Eligibility

Sex: ALLMin age: 4 Years

Medical Language ↔ Plain English

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.

Locations (10)

University of California, San Diego-Altman Clinical and Translational Research Institute

La Jolla, California, United States

Nationwide Childrens Hospital

Columbus, Ohio, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Outcomes

Primary Outcomes

Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers

Time frame: Baseline, Day 60

Secondary Outcomes

Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Blot

Time frame: Baseline, Day 60

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)

Time frame: Baseline, Day 60

Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)

Time frame: Baseline, Day 60

Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score