Vesleteplirsen for Duchenne Muscular Dystrophy

(MOMENTUM Trial)

No longer recruiting at 28 trial locations

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

Age: < 65

Sex: Male

Trial Phase: Phase 2

Sponsor: Sarepta Therapeutics, Inc.

Must be taking: Corticosteroids

Must not be taking: Exon 51-skipping therapy

Disqualifiers: Cardiac, Pulmonary, Hepatic, others

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

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

Compensation: Varies

Number of Visits: 8

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called vesleteplirsen (also known as SRP-5051) for Duchenne muscular dystrophy (DMD), a condition that weakens muscles over time. The study consists of two parts: Part A identified the safest dose, and Part B will further test this dose. Individuals with a genetic form of DMD treatable by skipping exon 51 in their DNA and who have been on stable steroid treatment can participate. The trial aims to ensure the treatment is safe and effective in managing DMD symptoms. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

The trial requires that you have been on a stable dose of oral corticosteroids for at least 12 weeks before starting the study, or not have taken them for at least 12 weeks. Also, you should not have started or changed the dose of certain medications like ACE inhibitors, β-blockers, or supplements within 12 weeks before the trial.

Is there any evidence suggesting that vesleteplirsen is likely to be safe for humans?

Research has shown that vesleteplirsen, also known as SRP-5051, has been studied for safety in people with Duchenne Muscular Dystrophy (DMD). Previous patients generally tolerated the treatment well, though some side effects were reported.

In one study, participants experienced mild to moderate side effects, including reactions at the infusion site and flu-like symptoms such as fever and chills. These side effects were temporary and usually resolved on their own.

Although the FDA has not yet approved vesleteplirsen for any condition, it has been tested in multiple trials, suggesting it is safe enough for ongoing testing. However, more research is needed to fully understand its long-term safety and effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Unlike the standard treatments for Duchenne Muscular Dystrophy, which primarily include corticosteroids and supportive therapies, vesleteplirsen offers a unique approach. Vesleteplirsen is an exon-skipping therapy designed to specifically target the genetic mutations that cause Duchenne Muscular Dystrophy, potentially slowing the disease's progression. Researchers are excited because this therapy is administered via intravenous infusion and could provide a more targeted, effective treatment option by addressing the root genetic cause rather than just managing symptoms.

What evidence suggests that vesleteplirsen might be an effective treatment for Duchenne Muscular Dystrophy?

Research has shown that vesleteplirsen, also known as SRP-5051, may effectively treat Duchenne Muscular Dystrophy (DMD). In one study, a dose of about 30 mg/kg of vesleteplirsen increased dystrophin production, a key protein for muscle function, to 5.17%. It also improved exon skipping, a process that helps create more functional dystrophin, to 11.11%. These findings suggest that vesleteplirsen could enhance muscle health in people with DMD. Early results from previous studies were positive, indicating its potential as a valuable treatment option. Participants in this trial will receive vesleteplirsen in two parts: Part A involves escalating dose levels, and Part B involves doses selected based on Part A data. ^{1 4 5 6 7}

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 individuals with Duchenne Muscular Dystrophy who can potentially benefit from exon 51-skipping treatment. Participants must have previously received SRP-5051 in Part A of this study or in Study 5051-102 to be eligible.

Inclusion Criteria

- Has received prior SRP-5051 treatment in Part A of this study or in Study 5051-102

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part A: Multiple Ascending Dose

Participants receive escalating dose levels of vesleteplirsen every 4 weeks via IV infusion to determine the maximum tolerated dose

75 weeks

Every 4 weeks (in-person)

Part B: Dose Expansion

Participants receive vesleteplirsen at doses selected from Part A every 4 weeks via IV infusion

Up to 5 years

Every 4 weeks (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• SRP-5051

Trial Overview The study tests Vesleteplirsen (SRP-5051) in two parts: first, finding the highest dose patients can tolerate without severe side effects (Part A), and then checking how well different doses work (Part B).

How Is the Trial Designed?

Group I: Part B: Vesleteplirsen Experimental Treatment 1 Intervention

Participants received vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This included the participants who rolled over from Part A, as well as the additional participants who enrolled at the beginning of Part B.

Group II: Part A: Vesleteplirsen Experimental Treatment 1 Intervention

Participants received escalating dose levels of vesleteplirsen, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.

Who Is Running the Clinical Trial?

Sarepta Therapeutics, Inc.

Lead Sponsor

Trials

54

Recruited

34,000+

Citations

1. investorrelations.sarepta.com investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-positive-data-part-b-momentum

Press Release - Investor Relations - Sarepta Therapeutics At target dose (~30 mg/kg), SRP-5051 dosed monthly resulted in mean dystrophin expression of 5.17% and mean exon skipping of 11.11% at 28 ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04004065

NCT04004065 | Two-Part Study for Dose Determination of ... Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11231654/

From FDA-approved drugs to peptide-conjugated ASO - PMC This article provides a comprehensive review and discussion of the available data pertaining to adverse reactions and toxicology associated with FDA-approved ...

4. parentprojectmd.org parentprojectmd.org/sarepta-therapeutics-reports-positive-clinical-results-from-phase-2-momentum-study-of-srp-5051-in-patients-with-duchenne-amenable-to-skipping-exon-51/

Sarepta Therapeutics Reports Positive Clinical Results ... Sarepta has announced positive results from Part A of the MOMENTUM study (Study 5051-201), a Phase 2 clinical trial of SRP-5051, its next-generation treatment ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03375255

NCT03375255 | A Study to Evaluate the Safety, Tolerability ... The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as ...

6. parentprojectmd.org parentprojectmd.org/sarepta-announces-discontinuation-of-srp-5051-development-for-duchenne/

Sarepta Announces Discontinuation of SRP-5051 ... Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta's PPMO chemistry and exon-skipping technology for individuals ...

7. investorrelations.sarepta.com investorrelations.sarepta.com/static-files/6904f0f5-b57a-469c-ae6d-42bdc09d44f6

Cohort of MOMENTUM Study of SRP-5051 for Duchenne ... Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients. Acta Myologica. 2012;31(1): 121-125. Page 6. © ...