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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 26 and at week 50
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ week 26 and at week 50
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 26 and at week 50
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531
Secondary outcome measures
North Star Ambulatory Assessment (NSAA) (Version 2.0), including time to stand and a timed 10-meter walk/run, with a range of 0 to 34 where higher scores indicate better outcome.
Performance of the Upper Limb (PUL) (Version 2.0) with a range of 0 to 64 where higher scores indicate a better outcome.
Stride Velocity 95th Centile (SV95C)/upper limb outcome (non-ambulatory patients)
Trial Design
1Treatment groups
Experimental Treatment
Group I: WVE-N531Experimental Treatment1 Intervention
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Who is running the clinical trial?
Wave Life Sciences Ltd.Lead Sponsor
11 Previous Clinical Trials
467 Total Patients Enrolled
Medical Director, MDStudy DirectorWave Life Sciences
76 Previous Clinical Trials
16,233 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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