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Exon Skipping Agent

WVE-N531 for Duchenne Muscular Dystrophy

Phase 1 & 2
Waitlist Available
Research Sponsored by Wave Life Sciences Ltd.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ambulatory or non-ambulatory male
Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention
Must not have
Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug for Duchenne muscular dystrophy, to see if it is safe and effective.

Who is the study for?
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.
What is being tested?
The 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.
What are the potential side effects?
Since this summary does not include specific side effects from the provided information, generally speaking, potential side effects may range from injection site reactions to more systemic responses like fatigue or gastrointestinal issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a man, able to walk or not.
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My DMD gene mutation can be treated with exon 53 targeting.
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I completed Part A and waited at least 18 weeks before starting Part B.
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I can move my shoulders somewhat.
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I have been diagnosed with Duchenne Muscular Dystrophy based on my symptoms.
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My heart and lungs are functioning well, meeting the study's required levels.
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I have enough muscle mass for biopsy procedures.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I haven't had major surgery in the last 3 months and don't plan any during the study.

Timeline

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

1Treatment groups
Experimental Treatment
Group I: WVE-N531Experimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

Wave Life Sciences Ltd.Lead Sponsor
12 Previous Clinical Trials
484 Total Patients Enrolled
Medical Director, MDStudy DirectorWave Life Sciences
80 Previous Clinical Trials
16,481 Total Patients Enrolled
~0 spots leftby Jan 2025
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