WVE-N531 for Duchenne Muscular Dystrophy (FORWARD-53)
This study is testing WVE-N531, an antisense oligonucleotide (ASO), for Duchenne Muscular Dystrophy (DMD). WVE-N531 is given intravenously (through a vein). The study aims to understand how safe WVE-N531 is, how well your body handles it, and its effects on DMD. To join, you must be a male between 4 and 18 years old with a confirmed DMD diagnosis and a specific genetic change (mutation) that can be helped by an exon 53 intervention. The study measures safety and changes in dystrophin levels (a protein missing in DMD) in muscle tissue. Parts A and B are complete, and all patients in Part B chose to continue treatment. Part C is now enrolling up to 15 new patients.
- Study design
- This is an open-label study, meaning you and your doctors will know if you are receiving WVE-N531. It is designed to enroll up to 26 participants.
- What's involved
- Participants will receive WVE-N531 every two or four weeks. You will have muscle biopsies at the beginning and after 24 weeks of treatment, and potentially after 48 weeks.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety will be monitored for up to 10 months after your last dose of WVE-N531.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
At a glance
Conditions
Where it's being run
5 sites across 4 statesStudy leadership
- Medical Director, MD · STUDY_DIRECTOR · Wave Life Sciences
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- Part A: Safety: Proportion of patients with adverse events (AEs)Day 1 (initial dose) up to 24 weeks after the last dose of Part A
- Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531At Week 26 and at Week 50 of Part B
- Part C: Pharmacodynamics: Change from baseline dystrophin level (% normal dystrophin) as assessed by a validated assay analysis in muscle tissue following multiple doses of WVE-N531At Baseline and following 24 weeks of treatment in Part C