Gene Transfer with SRP-6004 for Limb-Girdle Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial is testing the safety of a medicine called SRP-6004, given through a vein, in people who can walk but have a specific type of muscular dystrophy (LGMD2B/R2).

Eligibility Criteria

This trial is for ambulatory individuals with Limb Girdle Muscular Dystrophy, Type 2B/R2. Participants must have specific genetic mutations related to the condition, be able to perform motor tests, and not have high levels of certain antibodies. They can't join if they've had recent gene therapy or other investigational treatments, significant health issues, or need chronic drug treatment that could pose risks.

Inclusion Criteria

I have specific genetic mutations in the DYSF gene.

My arm and leg muscles are suitable for biopsy.

I can participate in tests that measure my muscle movements.

+2 more

Exclusion Criteria

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.

Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.

Participant Groups

The study is testing SRP-6004's safety when given through an IV infusion to people who can walk but have LGMD2B/R2. It aims to understand how well participants tolerate this potential new treatment and its effectiveness in treating their muscular dystrophy.

1Treatment groups

Experimental Treatment

Group I: SRP-6004Experimental Treatment1 Intervention

Participants will receive single IV infusion of SRP-6004 on Day 1.