Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
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.
Inclusion Criteria:

 -  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.

 Exclusion Criteria:

 -  Severe cardiomyopathy; cardiomyopathy that is managed by angiotensin-converting enzyme
 (ACE) inhibitors or beta blockers is acceptable provided the patient meets the LVEF
 inclusion criteria.

 -  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
 is longer.