Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: WVE-N531.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: No limits · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Jordan, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
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.
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
- Drug WVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
Primary outcome measures
- Number of participants with treatment related adverse events as assessed by the following parameters: [Time frame: Time Frame: Week 0 through Week 96]
Secondary outcome measures (4)
- North Star Ambulatory Assessment (NSAA) (Version 2.0) composite score, each item assessed using a 3 point scale, including: [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]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Jordan · 2 centers
- Istiklal Hospital/ Clinical Research Unit — Amman
- The Specialty Hospital (TSH)/ Advanced Clinical Center — Amman
United Kingdom · 1 center
- Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust — Headington
Identifiers
NCT: NCT07209332 · WVE-N531-002