Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

Trial ID or NCT#

NCT03508947

Status

not recruiting iconNOT RECRUITING

Purpose

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.

Official Title

A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy

Eligibility Criteria

Ages Eligible for Study: 5 Years to 18 Years
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * 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. 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:
  1. * 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.
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Investigator(s)

John W. Day, MD, PhD
John W. Day, MD, PhD
Neuromuscular neurologist, Neurophysiologist
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology

View on ClinicalTrials.gov