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Gene Therapy Trial Report

Summary

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)


NCTID NCT05906251 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Limb Girdle Muscular Dystrophy, Type 2B/R2
Disease Ontology Term DOID:0110276
Compound Name SRP-6004
Compound Description rAAVrh74-MHCK7-DYSF
Sponsor Sarepta Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 2 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant DYSF
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type dual AAVrh74
Editor Type none
Dose 1 2E12 vg
Dose 2 6E12 vg

Study Record Dates


Current Stage Phase1
Submit Date 2023-06-07
Completion Date 2025-06-13
Last Update 2025-09-04

Participation Criteria


Eligible Age 18 Years - 50 Years
Standard Ages Adult
Sexes Eligible for Study ALL
Eligibility Criteria
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. Note: Other inclusion or exclusion criteria could apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links