Duchenne Muscular Dystrophy Clinical Trial
Official title:
An Open-label Phase 1b/2a Study of WVE-N531 in Patients With 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. This study has 2 parts, Part A and Part B. Part A is complete.
In Part B, the study will include up to 12 patients. All patients will receive WVE-N531 at 10 mg/kg every other week for 48 weeks. Muscle biopsies will be performed following 24 and 48 weeks of treatment. The primary endpoint is dystrophin protein levels and participants will also be evaluated for safety, tolerability, digital and functional endpoints. Safety monitoring will occur through 18 weeks after the last dose. ;
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