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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) | Clinical Trials | Clareo Health

TERMINATEDPHASE1

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)

An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)

NCT05906251•Sarepta Therapeutics, Inc.

View on ClinicalTrials.gov

Summary

The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).

Eligibility

Age

18 - 50 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic DYSF Deoxyribonucleic acid (DNA) gene mutations as documented prior to screening visits.
•Participants must be ambulatory per protocol specified criteria.
•Ability to cooperate with motor assessment testing.
•Has accessible and intact lower and upper extremity musculature for biopsy.
•Have adeno-associated virus rhesus serotype 74 (rAAVrh74) antibody titers \< 1:400 (that is, not elevated) as determined by enzyme-linked immunosorbent assay (ELISA).

Exclusion Criteria

•Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
•Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
•Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Locations (1)

Nationwide Children's Hospital

Columbus, Ohio, United States

Key Dates

Start Date

May 22, 2023

Primary Completion Date

June 13, 2025

Completion Date

June 13, 2025

Last Updated

September 4, 2025

Enrollment

ACTUAL participants

Conditions

Limb Girdle Muscular Dystrophy

Interventions

SRP-6004

GENETIC

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

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)

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

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