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

Enrolling by invitation · Phase 2

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT07209332

Run by

Wave Life Sciences USA, Inc.

People needed

175

Starts

2025-12-28

Expected to finish

2029-03-01

Last updated by the study team

2026-03-06

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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