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WVE-N531 · 2 trials · 1 indication
* 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)
| Arm | Type | Description |
|---|---|---|
| WVE-N531 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| WVE-N531 | DRUG | WVE-N531 is an antisense oligonucleotide (ASO) |
Inclusion Criteria: 1. Participated in a prior study of WVE-N531 and has not experienced any significant toxicities due to WVE-N531 or significant clinical deterioration of general health since the last dose or early discontinuation visit. 2. Of note: if there will be a greater than 31-day gap betw...
WVE-N531 is an investigational therapy being developed for Duchenne Muscular Dystrophy, a genetic muscle-wasting disease. It is designed for patients whose disease is amenable to exon 53 skipping, and it carries a Rare Pediatric Disease designation from the FDA. It is in Phase 2 clinical development and has not been approved for any use.
WVE-N531 targets DMD exon 53. By directing exon 53 skipping in the dystrophin gene transcript, the therapy aims to restore a shortened but functional form of dystrophin in patients with Duchenne Muscular Dystrophy whose mutations are compatible with skipping this exon. Trial eligibility is biomarker selected around this exon 53 population.
WVE-N531 is being developed by Wave Life Sciences Ltd., which trades on Nasdaq under the ticker WVE. The company is running the clinical program in Duchenne Muscular Dystrophy, including an open-label Phase 1 study and a Phase 2 open-label extension study.
WVE-N531 is in Phase 2 clinical development for Duchenne Muscular Dystrophy. It is investigational and has not been approved by the FDA. The program includes a Phase 1 open-label study and a Phase 2 open-label extension study, and the FDA has granted it a Rare Pediatric Disease designation.
WVE-N531 has two registered clinical trials. NCT04906460 is the Phase 1 open-label FORWARD-53 study in patients with Duchenne Muscular Dystrophy, active but not recruiting, with 25 participants across the United States, Jordan, and the United Kingdom. NCT07209332 is a Phase 2 open-label extension study enrolling by invitation in Jordan and the United Kingdom with 175 participants.
The WVE-N531 trials enroll male patients with Duchenne Muscular Dystrophy. The Phase 1 FORWARD-53 study accepts participants aged 4 years and older, and neither study includes healthy volunteers. Both are open-label and uncontrolled, and the Phase 2 extension study is enrolling by invitation only.