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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| WVE-N531 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| WVE-N531 | Drug | WVE-N531 is an antisense oligonucleotide (ASO) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment related adverse events as assessed by the following parameters: |
| Time Frame: Week 0 through Week 96 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | Wave Life Sciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Istiklal Hospital/ Clinical Research Unit | Amman | Jordan | ||||
| The Specialty Hospital (TSH)/ Advanced Clinical Center |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| 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 |
| Amman |
| Jordan |
| Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust | Headington | Oxford | OX3 9DU | United Kingdom |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |