Gene Therapy for Limb-Girdle Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Sarepta Therapeutics, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests SRP-9003, a gene therapy, in patients with LGMD2E, a type of muscular dystrophy. The therapy uses a harmless virus to deliver a healthy gene into muscle cells to fix the genetic issue. SRP-9003 is a gene therapy that uses a harmless virus to deliver a healthy gene into muscle cells, a method previously demonstrated to be effective in treating muscular dystrophies.

Eligibility Criteria

This trial is for males and females with Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E), who have specific gene mutations and experience difficulty in physical activities like running or climbing stairs. Participants must be able to complete a 100 meter walk/run test at a minimum of 40% efficiency compared to healthy individuals.

Inclusion Criteria

I can walk or run 100 meters at least 40% as well as others my age and size.

I am of any gender and ethnicity.

I have trouble running, jumping, or climbing stairs.

+1 more

Exclusion Criteria

I don't have any illnesses or need for ongoing medication that would make gene therapy risky.

My heart's pumping ability is below 40%.

I have been diagnosed with or am being treated for an autoimmune disease.

+3 more

Participant Groups

The study tests SRP-9003 (bidridistrogene xeboparvovec), which is a new gene therapy delivered once to patients with LGMD2E. It's an open-label trial, meaning both the researchers and participants know what treatment is being given.

2Treatment groups

Experimental Treatment

Group I: Cohort 2: SRP-9003Experimental Treatment1 Intervention

Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.

Group II: Cohort 1: SRP-9003Experimental Treatment1 Intervention

Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.