Duchenne Muscular Dystrophy
Conditions
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.
Detailed description
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.
Interventions
DRUGWVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
Sponsors
Wave Life Sciences Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | * 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) |
| 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 | — |
Countries
Jordan, United Kingdom
Contacts
STUDY_DIRECTORMedical Director, MD
Wave Life Sciences
Outcome results
None listed