Wave Life Sciences is conducting a Phase 1b/2a clinical trial in Canada to evaluate the safety and tolerability of WVE-N531, a new exon-skipping investigational therapy.
WVE-N531 is an investigational antisense oligonucleotide (ASO) delivered by intravenous (IV) administration. Boys, ages 5-18 years, with Duchenne muscular dystrophy must have a mutation amenable to exon 53 skipping to participate. The study includes both ambulatory and non-ambulatory boys.
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 medication affects the body).
For further information, please contact Clinical Trial Concierge, Bonnie Wooten, at firstname.lastname@example.org or 1-866-226-8003 (toll-free).