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This study is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of ataluren 10, 10, 20 mg/kg in patients with nonsense mutation dystrophinopathy. Patients will be randomized in a 1:1 ratio to ataluren 10-, 10-, 20-mg/kg dose level or placebo. It is planned that 220 patients will be enrolled and patients will undergo 48 weeks of blinded treatment prior to the final analysis. Study assessments will be performed at clinic visits every 8 weeks. It is anticipated that an open-label extension study will be available to patients (who successfully complete the double-blind study) in countries where ataluren is not commercially available.
Who can participate in this study:
Ages Eligible for Study: 7 years to 16 years
Genders Eligible for Study: Male
Age ≥7 and ≤16 years
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists (CAP), the Clinical Laboratory Improvement Act/Amendment (CLIA) or an equivalent organization
Use of systemic corticosteroids (prednisone, prednisolone, or deflazacort) for a minimum of 6 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen (not related to body weight change) for a minimum of 3 months immediately prior to start of study
Screening 6-minute walk test. Patients need to be below the protocol-specified threshold for %-predicted 6MWD
Results of the 2 Baseline 6MWD results must be determined as valid and results of the Day 2 Baseline 6MWD must be within 20% of the Day 1 Baseline 6MWD
Baseline 6MWD (mean of valid Day 1 and Day 2 values) must be no more than a 20% reduction from the valid Screening 6MWD
Confirmed screening laboratory values within the central laboratory ranges (hepatic, renal, and serum electrolyte parameters)
Treatment with systemic aminoglycoside antibiotics within 3 months prior to start of study treatment
Initiation of systemic corticosteroids therapy within 6 months prior to start of study treatment
Change in systemic corticosteroid therapy (eg, change in type of drug, dose modification not related to body weight change, schedule modification, interruption, or reinitiation) within 3 months prior to start of study treatment
Any change (initiation, change in type of drug, dose modification, schedule modification,interruption, discontinuation, or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to start of study treatment
Ongoing use of coumarin-based anticoagulants (eg. warfarin), phenytoin, tolbutamide, or paclitaxel
Prior therapy with ataluren
Exposure to another investigational drug within 3 months prior to start of study treatment
History of major surgical procedure within 6 weeks prior to start of study treatment
Ongoing immunosuppressive therapy (other than corticosteroids)
Ongoing participation in any clinical trial (except for studies specifically approved by PTC Therapeutics)
Expectation of major surgical procedure (eg, scoliosis surgery) during the 12-month treatment period of the study
Requirement for daytime ventilator assistance. Note: Evening ventilator assistance and use of bi-level positive airway pressure (Bi-PAP) therapy is allowed
Uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D)
Who to contact:
If you are interested in this study, please contact the Research Coordinator, Susan Gailey, CCRC, phone: (614) 355-2897.
A description of the study is also listed at the website www.ClinicalTrials.gov with the identifier NCT01826487.