NCT07209332

Brief 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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
175

participants targeted

Target at P75+ for phase_2

Timeline
35mo left

Started Dec 2025

Typical duration for phase_2

Geographic Reach
2 countries

3 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress11%
Dec 2025Mar 2029

First Submitted

Initial submission to the registry

August 14, 2025

Completed
2 months until next milestone

First Posted

Study publicly available on registry

October 7, 2025

Completed
3 months until next milestone

Study Start

First participant enrolled

December 28, 2025

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2029

Last Updated

March 6, 2026

Status Verified

February 1, 2026

Enrollment Period

3.2 years

First QC Date

August 14, 2025

Last Update Submit

March 4, 2026

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

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

    * 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)

    Time Frame: Week 0 through Week 96

Secondary Outcomes (4)

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

    At baseline Week 0 through Week 96

  • 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)

    At baseline Week 0 through Week 96

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

    At baseline Week 0 through Week 96

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

    At baseline Week 0 through Week 96

Study Arms (1)

WVE-N531

EXPERIMENTAL
Drug: WVE-N531

Interventions

WVE-N531DRUG

WVE-N531 is an antisense oligonucleotide (ASO)

WVE-N531

Eligibility Criteria

Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Istiklal Hospital/ Clinical Research Unit

Amman, Jordan

Location

The Specialty Hospital (TSH)/ Advanced Clinical Center

Amman, Jordan

Location

Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust

Headington, Oxford, OX3 9DU, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Medical Director, MD

    Wave Life Sciences

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 14, 2025

First Posted

October 7, 2025

Study Start

December 28, 2025

Primary Completion (Estimated)

March 1, 2029

Study Completion (Estimated)

March 1, 2029

Last Updated

March 6, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)JO(2)