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

Target Gene/Variant	DYSF
Therapeutic Modality	Gene transfer
Therapy Route	in vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Gene Delivery System Type	Viral transduction
Vector Type	dual AAVrh74
Dose 1	2E12 vg
Dose 2	6E12 vg

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

No.of Trial Sites	1
Locations	United States