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Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
Sponsor: Wave Life Sciences Ltd.
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.
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
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
175
Start Date
2025-12-28
Completion Date
2029-03
Last Updated
2026-03-06
Healthy Volunteers
No
Conditions
Interventions
WVE-N531
WVE-N531 is an antisense oligonucleotide (ASO)
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