WVE-N531 Exon 53 Skipping Therapy for Duchenne Muscular Dystrophy: Open-Label Extension Study

Enrolling by InvitationPhase 2Exon Skipping

Age: 4–18 years
Phase: Phase 2
Sponsor: Wave Life Sciences
Therapeutic Approach: Exon Skipping
Variant Requirement: 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.
Eligible Sex: Male
Ambulation: Ambulatory and Non-Ambulatory
Study Start (Actual): 2025-12-28
Primary Completion (Estimated): 2029-03
Study Completion (Estimated): 2029-03
Enrollment (Estimated): 175
Countries: United KingdomJordan

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.

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.

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Study Requirements and Criteria