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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: 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.

• Study Type: Interventional
• Enrollment (Estimated): 175
• Phase: Phase 2

Contacts and Locations

Study Locations

• Jordan
  • Amman, Jordan
    • Istiklal Hospital/ Clinical Research Unit
  • Amman, Jordan
    • The Specialty Hospital (TSH)/ Advanced Clinical Center
• United Kingdom
  • Oxford
    • Headington, Oxford, United Kingdom, OX3 9DU
      • Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: WVE-N531	Drug: WVE-N531; WVE-N531 is an antisense oligonucleotide (ASO)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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: Week 0 through Week 96

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	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)		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

Collaborators and Investigators

• Sponsor: Wave Life Sciences Ltd.
• Investigators: Study Director: Medical Director, MD, Wave Life Sciences

Study record dates

Study Major Dates

• Study Start (Actual): December 28, 2025
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): March 1, 2029

Study Registration Dates

• First Submitted: August 14, 2025
• First Submitted That Met QC Criteria: October 3, 2025
• First Posted (Actual): October 7, 2025

Study Record Updates

• Last Update Posted (Actual): March 6, 2026
• Last Update Submitted That Met QC Criteria: March 4, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: WVE-N531-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes