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Enrolling by Invitation

NCT Number: NCT07209332

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

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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Istiklal Hospital/ Clinical Research Unit, Amman, Jordan

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • 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

  • 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.
  • 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.

Treatment and study plan

WVE-N531

Drug

WVE-N531 is an antisense oligonucleotide (ASO)

Primary outcomes

  1. Number of participants with treatment related adverse events as assessed by the following parameters:

    Time frame: 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 outcomes

  1. North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including:

    Time frame: 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)

  2. 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

  3. Stride Velocity 95th Centile (SV95C) (collected in 3 consecutive weeks)

    Time frame: At baseline Week 0 through Week 96

  4. Upper limb proximal strength (assessed by handheld myometer measured in Kilograms)

    Time frame: At baseline Week 0 through Week 96

Sponsors and collaborators

Lead sponsor

Wave Life Sciences USA, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
First posted
Oct 7, 2025
Registry last updated
Mar 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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