What is the mechanism of action of this treatment?

The most common treatments for Duchenne Muscular Dystrophy (DMD) include genetic therapies such as exon skipping drugs. These drugs, including eteplirsen, golodirsen, viltolarsen, and casimersen, work by skipping over specific exons in the DMD gene, allowing the production of a shorter but functional dystrophin protein. This is crucial for DMD patients because dystrophin is essential for muscle fiber stability and function. By increasing dystrophin production, these treatments aim to slow disease progression, improve muscle function, and enhance the quality of life for patients with DMD.

What side effects are associated with this type of intervention?

The most common treatments for Duchenne Muscular Dystrophy (DMD) include antisense oligonucleotides like viltolarsen, eteplirsen, golodirsen, and casimersen, as well as glucocorticoids. Known side effects of these antisense oligonucleotides include upper respiratory tract infections, injection site reactions, cough, fever, headache, and potential renal toxicity, which necessitates regular monitoring of kidney function. Glucocorticoids, such as prednisone and deflazacort, commonly cause weight gain, decreased linear growth, cushingoid appearance, delayed puberty, and increased risk of bone fractures. Regular monitoring and management of these side effects are essential for patients undergoing these treatments.

What prior FDA approvals exist?

Several exon-skipping drugs have received FDA approval for the treatment of Duchenne Muscular Dystrophy (DMD). Eteplirsen (ExonDys 51) targets exon 51 and is used to treat approximately 13% of DMD patients. Golodirsen (Vyondys 53) targets exon 53 and is suitable for about 8% of patients. Viltolarsen also targets exon 53 and has shown increased dystrophin production in clinical trials. Casimersen targets exon 45 and was approved based on its ability to increase dystrophin levels. These treatments aim to produce a shorter but functional dystrophin protein, similar to the approach being studied with Vesleteplirsen.

What other types of research exist?

Promising alternatives to Vesleteplirsen for DMD patients include Casimersen, Eteplirsen, Golodirsen, and Viltolarsen. These treatments are antisense oligonucleotides designed to induce exon skipping, which increases dystrophin production. Casimersen targets exon 45, Eteplirsen targets exon 51, Golodirsen targets exon 53, and Viltolarsen also targets exon 53. These therapies have shown efficacy in increasing dystrophin levels and have been granted accelerated approval by the FDA, with ongoing studies to confirm clinical benefits.

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Exon Skipping Agent

Vesleteplirsen for Duchenne Muscular Dystrophy (MOMENTUM Trial)

Phase 2

Waitlist Available

Research Sponsored by Sarepta Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests vesleteplirsen, a drug, for safety and tolerance. It involves participants from previous studies and new ones. The goal is to find the highest safe dose and monitor side effects.

Who is the study for?

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.

What is being tested?

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).

What are the potential side effects?

While specific side effects are not listed, typically such trials monitor for reactions at injection sites, potential kidney or liver issues, blood changes, muscle pain, and general discomfort.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Part B: VesleteplirsenExperimental Treatment1 Intervention

Participants will receive vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This includes the participants who rollover from Part A, as well as the additional participants who will be enrolled at the beginning of Part B.

Group II: Part A: VesleteplirsenExperimental Treatment1 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.

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Duchenne Muscular Dystrophy (DMD) include genetic therapies such as exon skipping drugs. These drugs, including eteplirsen, golodirsen, viltolarsen, and casimersen, work by skipping over specific exons in the DMD gene, allowing the production of a shorter but functional dystrophin protein. This is crucial for DMD patients because dystrophin is essential for muscle fiber stability and function. By increasing dystrophin production, these treatments aim to slow disease progression, improve muscle function, and enhance the quality of life for patients with DMD.