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