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)

Sarepta Therapeutics

Status and phase

Active, not recruiting

Phase 1

Conditions

Limb Girdle Muscular Dystrophy

Treatments

Genetic: SRP-6004

Study type

Interventional

Funder types

Industry

Identifiers

NCT05906251

SRP-6004-102

Details and patient eligibility

About

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).

Enrollment

2 patients

Sex

All

Ages

18 to 50 years old

Volunteers

No 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.

Note: Other inclusion or exclusion criteria could apply.