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Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
study id #: NCT03179631
condition: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
This study is a long-term study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy.
intervention: Ataluren, PLACEBO
mechanism of action: Stop codon read through to promote dystrophin production
start date: July 6, 2017
estimated completion: March 31, 2020
phase of development: Phase 3
size / enrollment: 250
This study is a randomized, double-blind, placebo-controlled, 72-week study followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Patients will be randomized in a 1:1 ratio to ataluren or placebo. Patients will receive blinded study drug TID at morning, midday, and evening for 72 weeks, after which all patients will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period. The total sample size of ~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary analysis population (age 7 to 16 years old, baseline 6MWD >=300 meters, supine to stand >=5 seconds). The study will be conducted in the United States and other countries around the world.
- 6- Minute Walk Test [Time Frame: 72 weeks]
- Timed Function Tests [Time Frame: 72 weeks]
- North Star Ambulatory Assessment [Time Frame: 72 weeks ]
- Performance of Upper Limb (in patients >=7 years old at baseline) [Time Frame: 72 weeks]
- Myometry (in patients <7 years old at baseline) [Time Frame: 72 weeks]
- Magnetic Resonance Imaging (MRI) (at pre-qualified sites) [Time Frame: 72 weeks]
- EQ-5D [Time Frame: 72 weeks]
- Ataluren safety profile characterized by adverse events and abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms [Time Frame: 72 weeks]
• Male sex.
• Age >=5 years.
• Phenotypic evidence of DMD
• 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.
• Prior or 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.
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