A Phase 2 interventional study of WVE-N531 in Duchenne Muscular Dystrophy, sponsored by Wave Life Sciences USA, Inc.. Enrolling by invitation at 3 sites in 2 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2026-03-06.
Sponsored by Wave Life Sciences USA, Inc. · Phase 2, Interventional, and Treatment
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531 in patients with DMD who participated in another study of WVE-N531. All patients will have rolled over from a previous study of WVE-N531.
The study will include up to 175 patients from planned and ongoing WVE-N531 studies globally. All patients will continue to receive 10 mg/kg WVE-N531 IV every 4 weeks (Q4W), through Week 96. Safety monitoring will continue for approximately 11 months after the final dose. The primary objective of the study is to evaluate the long-term safety and tolerability of WVE-N531.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's planned enrollment of 175 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Wave Life Sciences USA, Inc. is the lead sponsor of 9 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: WVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
Number of participants with treatment related adverse events as assessed by the following parameters:
* Complete Physical examination, including height and weight * Vital Signs (via blood pressure, temperature and pulse) * Safety Laboratory Tests (including complete blood cell count, urinalysis and clinical chemistry) * ECG (12- Lead single tracing) * ECHO (including left ventricular ejection fraction) * Pulmonary Function Tests (including Peak Flow Rate, Cough Peak Flow, Forced Vital Capacity and Maximum Inspiratory Pressure)
Time frame: Time Frame: Week 0 through Week 96
North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including:
* Time to stand (measured in seconds ) * Timed 10-meter walk/run (measured in seconds) * Four-stair climb time (measured in time to climb 4 stairs in seconds)
Time frame: At baseline Week 0 through Week 96
Performance of the Upper Limb (PUL) (Version 2.0) (measured by total score and score of high level shoulder dimension, Mid-level Elbow Dimension, Distal Wrist and Hand Dimension)
Time frame: At baseline Week 0 through Week 96
Stride Velocity 95th Centile (SV95C) (collected in 3 consecutive weeks)
Time frame: At baseline Week 0 through Week 96
Upper limb proximal strength (assessed by handheld myometer measured in Kilograms)
Time frame: At baseline Week 0 through Week 96
Plan to share: No
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for Mar 2029, 39 months after the start.
Of 40 industry-sponsored phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 42. This study's planned 39 months is above that.
Counting every sponsor, half of 47 studies reached it within 31 months; a quarter within 20 and three quarters within 42.
How these figures are worked out, and which studies are left out of them.
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Muscular Dystrophy, Duchenne →
Wave Life Sciences USA, Inc.