Gene Therapy for Limb-Girdle Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+1 other location

Age: < 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?The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.

Eligibility Criteria

This trial is for individuals aged 4-50 with Limb Girdle Muscular Dystrophy, type 2E/R4 who are either non-ambulatory (can't walk) or ambulatory (can walk). They must have specific genetic mutations and be able to undergo muscle testing. Those with significant other illnesses, exposure to gene therapy, or conditions affecting protocol compliance cannot participate.

Inclusion Criteria

I am between 4 and 50 years old and cannot walk.

I have specific genetic mutations in the β-sarcoglycan gene.

I can participate in muscle strength tests.

+1 more

Exclusion Criteria

Presence of any other clinically significant illness or medical condition, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, neuromuscular (other than LGMD2E/R4), or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability

I haven't had gene therapy or experimental drugs within the trial's time limits.

I cannot take corticosteroids due to health reasons.

Participant Groups

The study tests the safety and effectiveness of SRP-9003, a gene transfer treatment aimed at expressing β-SG in skeletal muscles of LGMD2E/R4 patients. It includes two groups: one that can walk (Cohort 1) and one that cannot (Cohort 2), assessing how well the treatment works in each.

1Treatment groups

Experimental Treatment

Group I: SRP-9003Experimental Treatment1 Intervention

Participants will receive single IV infusion of SRP-9003 on Day 1.