WVE-N531 for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+3 other locations

Age: < 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Wave Life Sciences Ltd.

No Placebo Group

Trial Summary

What is the purpose of this trial?

This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 2 parts, Part A and Part B. Part A is completed. Part B is completed. Following completion of Part B, all patients have elected to continue to receive study drug in an optional open-label extension arm.

Research Team

Medical Director, MD

Principal Investigator

Wave Life Sciences

Eligibility Criteria

This trial is for patients with Duchenne muscular dystrophy (DMD) who have a specific mutation in the DMD gene that can be targeted by exon 53 skipping. Participants should show certain levels of muscle function, have stable breathing capacity, and heart function within set ranges. They must also be on a consistent corticosteroid treatment for at least six months.

Inclusion Criteria

New patients may also be screened for Part B

I am a man, able to walk or not.

My DMD gene mutation can be treated with exon 53 targeting.

See 6 more

Exclusion Criteria

Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the study procedures

I haven't had major surgery in the last 3 months and don't plan any during the study.

Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit

Treatment Details

Interventions

WVE-N531 (Exon Skipping Agent)

Trial OverviewThe study is testing WVE-N531, administered intravenously to see if it's safe and how it affects the body (pharmacokinetics) and disease symptoms (pharmacodynamics). The trial has two parts; Part A is finished. It aims to understand how this drug could help manage Duchenne muscular dystrophy.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: WVE-N531Experimental Treatment1 Intervention