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

Not Applicable

Not yet recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: WVE-N531

• Registration Number: NCT07209332
• Lead Sponsor: Wave Life Sciences Ltd.

Brief 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 occur 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.

Study Timeline

• Study First Submitted: 2025-08-14
• Status Verified: 2025-09
• Study First Submitted QC: 2025-10-03
• Last Update Submitted: 2025-10-03
• Study First Posted: 2025-10-07
• Last Update Posted: 2025-10-07
• Study Start: 2025-11-01
• Primary Completion: 2029-03
• Study Completion: 2029-03-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: Male
• Target Recruitment: 175

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 between the last dose on the prior study and the FD on this study, the case should be discussed between the Investigator and Medical Monitor.

Exclusion Criteria

1. Any clinically significant medical finding or change during or following participation in the prior WVE-N531 study, other than DMD that, in the judgment of the Investigator, would affect the potential safety of the patient to receive WVE-N531 or interfere with participation in the study.
2. Any recreational substance use (including prescribed cannabinoids), with the exception of alcohol and nicotine, irrespective of legality, within 2 months prior to FD and/or unwilling to refrain from such use for the duration of the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
WVE-N531	WVE-N531	-

Primary Outcome Measures

Name	Time	Method
Number of participants with treatment related adverse events as assessed by the following parameters:	Time Frame: Week 0 through Week 96	* 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)

Secondary Outcome Measures

Name	Time	Method
North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including:	At baseline Week 0 through Week 96	* 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)
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)	At baseline Week 0 through Week 96
Stride Velocity 95th Centile (SV95C) (collected in 3 consecutive weeks)	At baseline Week 0 through Week 96
Upper limb proximal strength (assessed by handheld myometer measured in Kilograms)	At baseline Week 0 through Week 96

Trial Locations

Locations (3)

Istiklal Hospital/ Clinical Research Unit; 🇯🇴 Amman, Jordan

The Specialty Hospital (TSH)/ Advanced Clinical Center; 🇯🇴 Amman, Jordan

Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust; 🇬🇧 Headington, Oxford, United Kingdom