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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called SRP-9005, an experimental therapy for individuals with limb-girdle muscular dystrophy type 2C/R5, a condition that weakens muscles around the hips and shoulders. Researchers aim to determine if a single dose of SRP-9005 is safe and effective in both children and adults with this condition. Individuals who can walk unaided or possess specific genetic markers related to this condition might be eligible. As a Phase 3 trial, this represents the final step before FDA approval, offering participants an opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, it does mention that you cannot have taken any investigational medication within 6 months of the screening visit.

Is there any evidence suggesting that SRP-9005 is likely to be safe for humans?

Research has shown that SRP-9005, a gene therapy for a type of muscular dystrophy, was tested in mice. These studies found the treatment promising in terms of safety and effectiveness. However, safety concerns arose during human trials. Due to these concerns, the FDA paused Sarepta's trials, indicating the need for more research to fully understand the treatment's safety for people.

Since SRP-9005 is in a late-stage trial, earlier studies indicated it was somewhat safe for humans. However, the FDA's caution is important to consider. Participants should weigh these factors when deciding whether to join the study. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

SRP-9005 is unique because it offers a new approach to treating Limb-Girdle Muscular Dystrophy (LGMD) by using gene therapy. While current treatments mainly focus on managing symptoms and improving quality of life, SRP-9005 aims to address the underlying genetic cause of the disease. Researchers are excited about SRP-9005 because it involves a single intravenous infusion that has the potential to provide lasting benefits by directly targeting the genetic mutations responsible for LGMD. This approach could significantly change the way we treat this condition, offering hope for more effective and long-term solutions.

What evidence suggests that SRP-9005 might be an effective treatment for limb-girdle muscular dystrophy?

Research has shown that SRP-9005, which participants in this trial will receive, could help treat limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). Earlier studies found that it improved muscle function in mice. This treatment is a type of gene therapy, delivering a healthy version of the gene that malfunctions in LGMD2C/R5. Early results suggest this method can restore muscle strength and slow the disease's progression. There is significant hope that SRP-9005 could offer real benefits for people with this condition. ^{1 2 3 4 6}

Who Is on the Research Team?

Medical Director

Principal Investigator

Sarepta Therapeutics, Inc.

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06952686

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy ... 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).

2. investorrelations.sarepta.com investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-pipeline-progress-multiple-limb

Press Release - Investor Relations - Sarepta Therapeutics SRP-9005 is an investigational gene therapy for patients with limb-girdle muscular dystrophy Type 2C (LGMD2C/R5), also known as gamma- ...

3. institut-myologie.org institut-myologie.org/en/2023/04/12/gene-therapy-confirms-its-efficacy-in-the-mouse-model-of-limb-girdle-muscular-dystrophy-related-to-sgcg/

Gene therapy confirms its efficacy in the mouse model ... A study investigated the safety and efficacy of SRP-9005 (rAAVrh74 vector) when injected intravenously into the mouse model of GIST-related ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9929442/

Systemic γ-sarcoglycan AAV gene transfer results in dose ... Gene transfer therapy has emerged as a promising treatment for monogenic diseases, including limb-girdle muscular dystrophy 2C/R5 (LGMD 2C/R5). Systemic ...

5. ctv.veeva.com ctv.veeva.com/study/a-study-of-srp-9005-in-limb-girdle-muscular-dystrophy-type-2c-r5-pediatric-and-adult-participants

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type ... 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).

6. fiercebiotech.com fiercebiotech.com/biotech/sarepta-ldmd-trials-all-hit-fda-hold-amid-newly-surfaced-safety-concerns

FDA slaps clinical hold across all of Sarepta's LGMD trials The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company's investigational limb girdle muscular dystrophy