A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLEDEFFICACY AND SAFETY STUDY OF ATALUREN INPATIENTS WITH NONSENSE MUTATION DUCHENNE MUSCULARDYSTROPHY AND OPEN-LABEL EXTENSIO

PTC Therapeutics Inc0 个研究点目标入组 68 人开始时间: 待定最近更新: 2024年6月24日

研究概览

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•Age 5 years and older
•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 greater than or equal to 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.

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