WVE-N531: WVE-N531 is an antisense oligonucleotide (ASO)
Study 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.
Eligibility
Sex
MALE
Min age
—
Max age
—
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.
Primary outcome measure(s)
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)
Trial sites (3)
Facility
City
Region
Status
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
More Wave Life Sciences USA, Inc. trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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