Trial Search Results
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
Stanford is currently accepting patients for this trial.
Wave Life Sciences Ltd.
- Drug: WVE-210201
- Drug: Placebo
- Diagnosis of Duchenne muscular dystrophy (DMD) based on clinical phenotype with
increased serum creatine kinase
- Documented mutation in the Dystrophin gene associated with DMD that is amenable to
exon 51 skipping
- Ambulatory or non-ambulatory male patients aged ≥5 - ≤18 years
- Stable pulmonary and cardiac function as measured by:
1. Reproducible percent predicted forced vital capacity (FVC) ≥50%
2. Left ventricular ejection fraction (LVEF) >55% in patients <10 years of age and
>45% in patients ≥10 years of age, as measured (and documented) by echocardiogram
within one year prior to enrollment into the study.
- Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme
(ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF
- Need for mechanical or non-invasive ventilation OR anticipated need for mechanical or
non-invasive ventilation within the next year, in the opinion of the Investigator.
- Changes in nutritional or herbal supplements or concomitant medications within 1 month
prior to Screening visit or plans to modify dose or regimen during the study.
- Currently on anticoagulants or antithrombotics.
- Received treatment with eteplirsen or ataluren within the past 14 weeks.
- Received prior treatment with drisapersen.
- Received any investigational drug within the past 3 months or 5 half-lives, whichever
Ages Eligible for Study
5 Years - 18 Years
Genders Eligible for Study