Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Trial ID or NCT#



recruiting iconRECRUITING


This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Official Title

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Eligibility Criteria

Ages Eligible for Study: Older than 5 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - Male sex - Age ≥5 years - Phenotypic evidence of Duchenne Muscular Dystrophy - Nonsense point mutation in the dystrophin gene - Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment - 6MWD ≥150 meters - Ability to perform timed function tests within 30 seconds - Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria:
  1. - Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment. - Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy. - Prior or ongoing therapy with ataluren. - Known hypersensitivity to any of the ingredients or excipients of the study drug - Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial. - History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period. - Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy. - Uncontrolled clinical symptoms and signs of congestive heart failure - Elevated serum creatinine or cystatin C at screening.


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

Contact us to find out if this trial is right for you.


Monica Sangco