Long-term study of WVE-N531 in people with Duchenne muscular dystrophy
Tests the drug WVE-N531 over the long term in people with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Key facts
- Where
- Headington, United KingdomSee the site
- Phase
- Phase 2
Where in United Kingdom
- Oxford Children's Hospital, Oxford University Hospitals NHS Foundation TrustHeadington, United KingdomNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Wave Life Sciences USA, Inc.
- Trial code
- NCT07209332
- Record updated
- 6 March 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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: 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.