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Clinical Trials in the UK / NCT07209332
Enrolling by invitation Phase 2

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

NCT07209332 · tracked via the Priya Life Science UK tracker
Sponsor
Wave Life Sciences USA, Inc
Phase
Phase 2
Started
2025-12-28
Last updated
2026-03-06

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

WVE-N531 →

WVE-N531: WVE-N531 is an antisense oligonucleotide (ASO)

Study 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.

Eligibility

Sex
MALE
Min age
—
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

  • 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)

Trial sites (3)

FacilityCityRegionStatus
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

More Wave Life Sciences USA, Inc. trials in the UK

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD Recruiting
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Active, not recruiting
A Phase 1/2a Study of WVE-007 in Adults Living With Overweight or Obesity Recruiting

Other trials for the same condition

Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old Recruiting
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) Active, not recruiting
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients Active, not recruiting
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Active, not recruiting
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07209332 on ClinicalTrials.gov ↗ ← All trials in the UK