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

• Research type

  Research Study

• Full title

  An Open-label Extension Study to Investigate the Long-term Safety, Tolerability, Pharmacokinetics, and Efficacy of WVE-N531 in Patients with Duchenne Muscular Dystrophy Who Participated in Another Study of WVE-N531

• IRAS ID

  1012841

• Contact name

  Melanie Govignon

• Contact email

  mgovignon@wavelifesci.com

• Sponsor organisation

  Wave Life Sciences UK Limited

• Clinicaltrials.gov Identifier

  NCT07209332

• Research summary

  Duchenne muscular dystrophy (DMD) is caused by a mutation (a change) in the gene that contains the instructions for making a protein called dystrophin. Dystrophin protects muscles from stress and damage, and people with DMD are not able to make enough to protect their muscles. DMD is a progressive genetic condition for which there is currently no approved DMD therapy in the UK that addresses the underlying genetic cause of the disease, for patients who have a specific type of mutation that could be helped by skipping exon 53 in the DMD gene.

  WVE-N531-002 is an open-label (not blinded) extension study of WVE-N531 (the ‘study drug’) in boys with Duchenne Muscular Dystrophy (DMD) who have previously participated in another study of the same study drug. This study aims to assess the safety, tolerability (the degree to which side effects of a drug can be tolerated), and various functional outcomes over a longer period. The study drug is designed to skip a specific part of the dystrophin gene called Exon 53. Skipping Exon 53 may allow the body to produce a shortened, but still working, form of dystrophin. This may result in improved muscle function in people with DMD.

  The study will involve up to 175 boys globally, all of whom would have participated in a previous study of WVE-N531. The boys will be given the study drug every 4 weeks, through Week 96. Safety monitoring will occur for approximately 11 months after the final dose.

• REC name

  Wales REC 3

• REC reference

  25/WA/0262

• Date of REC Opinion

  15 Oct 2025

• REC opinion

  Further Information Favourable Opinion