Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

NCT ID: NCT06747273

Last Updated: 2025-09-04

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-09
• Study Completion Date: 2025-06-18

Brief Summary

The primary objective of this study is to evaluate the safety of SRP-9004.

Conditions

Limb Girdle Muscular Dystrophy; Limb Girdle Muscular Dystrophy Type 2D/R3

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SRP-9004

Participants will receive a single dose of SRP-9004 on Day 1.

Group Type: EXPERIMENTAL

SRP-9004

Intervention Type: DRUG

Intravenous (IV) infusion.

Interventions

SRP-9004

Intravenous (IV) infusion.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Ambulatory participants, defined as able to walk without assistive aid, 10MWR <30 seconds, and NSAD total score ≥25; non-ambulatory participant, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3.
• Ambulatory participants must be 4 to 20 years of age and the non-ambulatory participant must be ≥4 years of age.
• All participants must be ≤70 kilograms
• Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening.
• Able to cooperate with muscle testing.
• Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).

Exclusion Criteria

• Left ventricular ejection fraction <40% or clinical signs and/or symptoms of cardiomyopathy
• FVC ≤40% of predicted value and/or requirement for nocturnal ventilation
• Any other clinically significant illness, including neuromuscular (other than limb girdle muscular dystrophy type 2D/R3 [LGMD2D/R3]), that in the opinion of the Investigator might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.

• Minimum Eligible Age: 4 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Nationwide Children's Hospital

Columbus, Ohio, United States

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Countries

United States

Other Identifiers

SRP-9004-102

Identifier Type: -

Identifier Source: org_study_id