Enrollment is still open for the Wave Life Sciences Phase 1b/2a clinical trial of WVE-N531, an investigational antisense oligonucleotide (ASO) delivered intravenously (directly to the vein).
Boys ages 5-18 years, with Duchenne muscular dystrophy, both walking or not walking and have a mutation amenable to exon 53 skipping are eligible to participate. Exon 53 skipping is applicable for patients with deletions in exons 45-52, 47-52, 48-52, 49-52, 50-52, and 52. If you are unsure of your deletion or if you qualify, please contact [email protected].
This is an open-label study, meaning all participants and investigators will know that the investigational therapy is being administered, and no participant will receive a placebo or dummy treatment. Along with safety and tolerability, the study seeks to understand pharmacokinetics (how the drug is absorbed and metabolized in the body) and pharmacodynamics (how the drug affects the body). Click the button below to learn more about the study.
Recruitment in Canada
The trial is currently recruiting patients at the London Health Sciences Centre in London, Ontario. If you are interested in participating in this trial, please contact Clinical Trial Concierge Bonnie Wooten at c[email protected] or at 1-866-226-8003 (toll-free).
If you are interested in learning about more clinical trials you may qualify for, please visit our Clinical Trial Finder Tool.