Phase 2 Muscular Dystrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- Patients who have been diagnosed with Duchenne muscular dystrophy (DMD) based on their symptoms and clinical assessment
- Patients who have a confirmed genetic change in the DMD gene that can be targeted by a specific type of treatment called "exon 53 intervention"
- Patients who have some remaining arm and shoulder movement, as measured by a specific upper limb test (a score of 1 or higher on certain shoulder items)
- Male patients who are either able to walk or unable to walk (Parts A and B), or male patients who are currently able to walk (Part C only)
- Patients whose lungs are working at 50% or more of the expected level, based on a breathing test
- Patients whose heart is pumping well enough, confirmed by a heart scan (echocardiogram or cardiac MRI) done within the last 6 months
- Patients who have enough muscle tissue available for a small muscle sample (biopsy), preferably from the shoulder area
- Patients who have been on a stable steroid medication for at least 6 months before screening, with no dose changes in the last 3 months
- Previous participants from Part A may also be eligible for Part B after an 18-week rest period from their last dose
Who may not be able to join:
- Patients who have another significant health condition (besides DMD) that the doctor believes could make participation unsafe or difficult
- Patients who have had major surgery within the 3 months before the study starts, or who are planning major surgery at any point during the study (Parts B and C)
- Patients who have had a diagnosed problem with alcohol, cannabis, or other substance use (except nicotine) within the past 6 months (Part B only)
- Patients who have used any recreational substances — including prescribed cannabis — within the past 2 months, or who are unwilling to avoid such use for the whole study (Part C only)
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Director, MD, Wave Life Sciences
Phone: 855-215-4687
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Part A: Safety: Proportion of patients with adverse events (AEs); Part B: Pharmacodynamics: Dystrophin level (% normal dystrophin) as assessed by Western blot of muscle tissue following multiple doses of WVE-N531; Part C: Pharmacodynamics: Change from baseline dystrophin level (% normal dystrophin) as assessed by a validated assay analysis in muscle tissue following multiple doses of WVE-N531
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.