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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 | Clinical Trials | Clareo Health

TERMINATEDPHASE1

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

A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States

NCT06747273•Sarepta Therapeutics, Inc.

View on ClinicalTrials.gov

Summary

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

Eligibility

Age

4 - No limit years

Sex

ALL

Healthy Volunteers

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.

Locations (2)

Nationwide Children's Hospital

Columbus, Ohio, United States

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Key Dates

Start Date

January 9, 2025

Primary Completion Date

June 18, 2025

Completion Date

June 18, 2025

Last Updated

September 4, 2025

Enrollment

ACTUAL participants

Conditions

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

Interventions

SRP-9004

DRUG

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

NCT05876780

Limb Girdle Muscular Dystrophy

View study

ACTIVE_NOT_RECRUITING

3 Year Follow up on ANO5 Patients

NCT05206617

Neuromuscular DiseasesLimb Girdle Muscular Dystrophy

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 4, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

3 Year Follow up on ANO5 Patients

A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)

Schulze Muscular Dystrophy Ability Clinical Study

Stem Cell Therapy in Limb Girdle Muscular Dystrophy

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C