SRP-9005 for Limb-Girdle Muscular Dystrophy

(COMPASS Trial)

Overseen By Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Sarepta Therapeutics, Inc.

Must not be taking: Cardiac medications, Growth factors

Disqualifiers: Infections, Cardiomyopathy, Gene therapy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Single dose

15 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy.

Research Team

Medical Director

Principal Investigator

Sarepta Therapeutics, Inc.

Eligibility Criteria

This trial is for pediatric and adult patients with Limb Girdle Muscular Dystrophy type 2C/R5. Participants must be able to walk (with or without aid), have specific scores on mobility tests, and carry certain genetic mutations. They should not have high levels of antibodies against the therapy vector.

Inclusion Criteria

My AAVrh74 antibody levels are not high.

I have specific genetic mutations in the gamma-SG gene.

I can walk on my own without help and meet specific walking and assessment scores.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous (IV) infusion of SRP-9005

Single dose

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Treatment Details

Interventions

• Corticosteroid
• SRP-9005

Trial Overview The study is testing SRP-9005, a new treatment for LGMD2C/R5. It involves a single systemic dose and will evaluate both safety and effectiveness in two parts: Part A for initial assessment, followed by Part B for further evaluation.

Participant Groups

Group I: SRP-9005 Experimental Treatment 2 Interventions

Participants will receive a single intravenous (IV) infusion of SRP-9005.

Who Is Running the Clinical Trial?

Sarepta Therapeutics, Inc.

Lead Sponsor

Trials

54

Recruited

34,000+