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)
Launched by SAREPTA THERAPEUTICS, INC. · Jun 7, 2023
Trial Information
Current as of June 22, 2025
Active, not recruiting
Keywords
ClinConnect Summary
This clinical trial is studying a treatment called SRP-6004 for people with a specific type of Limb Girdle Muscular Dystrophy, known as LGMD2B/R2. The main goal of the study is to see if this treatment is safe and tolerable when given through an IV (a small tube inserted into a vein) in participants who can still walk. The trial is currently active but not recruiting new participants.
To be eligible for this study, participants must have specific genetic mutations related to their condition, be able to walk, and have certain muscle areas available for testing. They should also not have elevated levels of a specific virus that could affect the treatment. Overall, participants can expect to undergo assessments to monitor their health and how they respond to the treatment. It's important for potential participants to discuss with their doctors if they meet the criteria and understand any other specific requirements for joining the trial.
Gender
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.
About Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc. is a leading biopharmaceutical company focused on developing innovative genetic medicines to transform the treatment of rare diseases, particularly those caused by genetic mutations. With a strong emphasis on advancing therapies for Duchenne muscular dystrophy (DMD) and other neuromuscular disorders, Sarepta utilizes cutting-edge technologies, including gene therapy and RNA-targeted approaches, to address unmet medical needs. The company is committed to rigorous clinical research and collaboration with the scientific community to deliver safe and effective treatment options, ultimately aiming to enhance the quality of life for patients and their families.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Columbus, Ohio, United States
Patients applied
Trial Officials
Medical Director
Study Director
Sarepta Therapeutics, Inc.
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported