Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy

  • participants needed
  • sponsor
    Wave Life Sciences Ltd.
Updated on 27 November 2022
dmd gene
duchenne muscular dystrophy


This is a Phase 1b/2a 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


The study will include approximately 15 patients. An initial cohort will receive ascending doses of WVE-N531. Up to 4 dose levels (administered ≥4 weeks apart) will be evaluated in order to select a dose level for further multiple dose evaluation. The initial patients will receive up to 3 additional doses every other week at that dose level. Additional patients will then be enrolled and dosed every other week at that level. All patients will receive a maximum of 7 total doses followed by a minimum 8 week safety monitoring period.

Condition Duchenne Muscular Dystrophy
Treatment WVE-N531
Clinical Study IdentifierNCT04906460
SponsorWave Life Sciences Ltd.
Last Modified on27 November 2022

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