Actively Recruiting
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Led by Wave Life Sciences Ltd. · Updated on 2025-12-15
26
Participants Needed
5
Research Sites
290 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase 1b/2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.
CONDITIONS
Official Title
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male patients aged 4 to 18 years
- Diagnosis of Duchenne muscular dystrophy based on clinical phenotype
- Documented mutation in the DMD gene amenable to exon 53 skipping intervention
- Score of 1 or higher on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL)
- Ambulatory or non-ambulatory male (Part B) or ambulatory male (Part C)
- Stable pulmonary function with percent predicted forced vital capacity (FVC) of 50% or higher
- Stable cardiac function with left ventricular ejection fraction (LVEF) above 55% for patients under 10 years old and above 45% for patients 10 years or older, measured within 6 months prior to enrollment
- Adequate muscle for open muscle biopsies, preferably deltoid
- On stable corticosteroid therapy initiated at least 6 months prior to screening with no dose changes in the 3 months before screening
You will not qualify if you...
- Any significant medical condition other than Duchenne muscular dystrophy that would make participation unsuitable
- Major surgery within 3 months before Day 1 or planned major surgery during the study
- Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months before screening (Part B)
- Any recreational substance use, including prescribed cannabinoids except nicotine, within 2 months before screening or unwillingness to refrain from use during the study (Part C)
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 5 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202-3500
Actively Recruiting
2
Rare Disease Research LLC
Atlanta, Georgia, United States, 30329
Actively Recruiting
3
Istiklal Hospital/ Clinical Research Unit
Amman, Jordan
Actively Recruiting
4
The Specialty Hospital (TSH)/ Advanced Clinical Center
Amman, Jordan
Actively Recruiting
5
Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust
Headington, Oxford, United Kingdom, OX3 9DU
Actively Recruiting
Research Team
C
Clinical Operations
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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