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

Completed · Phase 1 · Has a placebo group

Conditions studied: Duchenne Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT03508947

Run by

Wave Life Sciences USA, Inc.

People needed

36

Starts

2018-01-24

Expected to finish

2019-03-06

Last updated by the study team

2019-04-08

Who can join

Age: 5 and older, up to 18. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• Reproducible percent predicted forced vital capacity (FVC) ≥50%
• 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.

You may not qualify if…

• 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.

Where it is running

• Rare Disease Research, LLC. — Atlanta, Georgia, United States
• UZ Gent — Ghent, Belgium
• Universitaire Ziekenhuizen Leuven — Leuven, Belgium
• CHR de la Citadelle — Liège, Belgium
• London Health Sciences Centre - Hospital — London, Ontario, Canada
• Hôpital Armand Trousseau — Paris, France
• U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud — Messina, Italy
• U.O. Immunologia Pediatrica — Milan, Italy
• Radbound University Nijmegen Medical Care — Nijmegen, Netherlands
• University Hospitals Bristol NHS Foundation Trust — Bristol, United Kingdom
• Alder Hey Children's Hospital — Liverpool, United Kingdom
• Evelina London Children's Hospital — London, United Kingdom
• UCL Institute of Child Health & Great Ormond Street Hospital for Children — London, United Kingdom

Full record on ClinicalTrials.gov

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