NCT07209332

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

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

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 175 (estimated)
• Sponsor: Wave Life Sciences Ltd. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	WVE-N531	WVE-N531 is an antisense oligonucleotide (ASO)

Timeline

• Start date: 2025-12-28
• Primary completion: 2029-03-01
• Completion: 2029-03-01
• First posted: 2025-10-07
• Last updated: 2026-03-06

Locations

3 sites across 2 countries: Jordan, United Kingdom

Regulatory

• FDA-regulated drug study