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

  • STATUS
    Recruiting
  • participants needed
    15
  • sponsor
    Wave Life Sciences Ltd.
Updated on 27 November 2022
dmd gene
duchenne muscular dystrophy

Summary

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

Description

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.

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

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