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

Phase 2Enrolling By InvitationNCT07209332

Wave Life Sciences USA, Inc.175 enrolled

Overview

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.

The study will include up to 175 patients from planned and ongoing WVE-N531 studies globally. All patients will continue to receive 10 mg/kg WVE-N531 IV every 4 weeks (Q4W), through Week 96. Safety monitoring will continue for approximately 11 months after the final dose. The primary objective of the study is to evaluate the long-term safety and tolerability of WVE-N531.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

175

Conditions

Duchenne Muscular Dystrophy

Interventions

WVE-N531DRUG

WVE-N531 is an antisense oligonucleotide (ASO)

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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.

Locations (3)

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

Outcomes

Primary Outcomes

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: Time Frame: Week 0 through Week 96

Secondary Outcomes

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

* Time to stand (measured in seconds ) * Timed 10-meter walk/run (measured in seconds) * Four-stair climb time (measured in time to climb 4 stairs in seconds)

Time frame: 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)

Time frame: At baseline Week 0 through Week 96

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

Time frame: At baseline Week 0 through Week 96

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

Time frame: At baseline Week 0 through Week 96

Data from ClinicalTrials.gov

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