Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Trial ID or NCT#
Status
Purpose
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
- - 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.
- - 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.
Investigator(s)
Contact us to find out if this trial is right for you.
Contact
Monica Sangco
650-206-3180
View on ClinicalTrials.gov