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

NCT06246513 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: SRP-9003-3012022-503112-17-00
• Study Type: interventional
• Target: 17
• Locations: 6 countries
• Sites: 10

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).

Conditions

Limb-girdle Muscular Dystrophy

Keywords

Limb Girdle Muscular Dystrophy Type 2E/R4; LGMD2E/R4; Pediatric; SRP-9003; scAAVrh74.MHCK7.hSGCB; bidridistrogene xeboparvovec; Non-ambulatory; Ambulatory

Outcome Measures

Primary Outcomes (1)

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

  Baseline, Day 60

Secondary Outcomes (13)

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

  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)

  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)

  Baseline, Day 60

• Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score

  Baseline through Month 60

• Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score

  Baseline through Month 60

Study Arms (1)

SRP-9003

EXPERIMENTAL

Participants will receive a single intravenous (IV) infusion of SRP-9003.

Biological: SRP-9003; Drug: Glucocorticoid

Interventions

SRP-9003 BIOLOGICAL

Solution for single IV infusion

Also known as: scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec

SRP-9003

Glucocorticoid DRUG

Oral tablet (prophylactic)

Also known as: Prednisone or equivalent

SRP-9003

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Cohort 1, only ambulatory participants:
• Able to walk without assistive aid
• MWR \<30 seconds
• NSAD ≥25
• Cohort 2, only non-ambulatory participants:
• MWR ≥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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (10)

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

La Jolla, California, 92037, United States

Location

Nationwide Childrens Hospital

Columbus, Ohio, 43205, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of The King's Daughter

Norfolk, Virginia, 23507, United States

Location

University Hospital Leuven (UZ Leuven)

Leuven, Vlaams Brabant, 3000, Belgium

Location

NMRC Gent (UZ Gent)

Ghent, 9000, Belgium

Location

Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum

Essen, North Rhine-Westphalia, 45147, Germany

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Hospital Sant Joan de Deu

Barcelona, 8950, Spain

Location

Newcastle University

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Interventions

Glucocorticoids; Prednisone

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2024
• First Posted: February 7, 2024
• Study Start: January 15, 2024
• Primary Completion: March 4, 2025
• Study Completion (Estimated): November 30, 2029
• Last Updated: March 2, 2026

Record last verified: 2026-02

Locations

ES (1); IT (1); DE (1); BE (2); US (4); GB (1)