An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy

PTC Therapeutics, Inc.0 个研究点开始时间: 2021年6月24日最近更新: 2021年8月24日

适应症

试验速览

暂无简介。

•Males =6 months to <2 years of age
•Body weight =7.5 kilograms (kg)
•Diagnosis of duchenne muscular dystrophy (DMD) based on an elevated serum creatine kinase and genotypic evidence of dystrophinopathy.
•Documentation of the presence of a nonsense mutation of the dystrophin gene as determined by gene sequencing prior to enrollment.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Participation in any drug or device investigation or whose sibling is currently participating in a blinded portion of another ataluren study or received an investigational drug within three months prior to the Screening Visit or who anticipate participating in any other drug or device clinical investigation or receiving any other investigational drug within the duration of this study.
•Expectation of a major surgical procedure during the study period.
•Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
•Ongoing use of the following drugs:
•a. Systemic aminoglycoside therapy and/or intravenous (IV) vancomycin.
•b. Coumarin-based anticoagulants (for example, warfarin), phenytoin, tolbutamide, or paclitaxel.
•c. Inducers of UGT1A9 (for example, rifampicin), or substrates of OAT1 or OAT3 (for example, ciprofloxacin, adefovir, oseltamivir, aciclovir, captopril, furosemide, bumetanide, valsartan, pravastatin, rosuvastatin, atorvastatin, pitavastatin).

发起方