Vesleteplirsen for Duchenne Muscular Dystrophy

(MOMENTUM Trial)

Recruiting at 27 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

62 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Medical Director

Principal Investigator

Sarepta Therapeutics, Inc.

Eligibility Criteria

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

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

Treatment Details

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).

Participant Groups

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+