A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.0 个研究点目标入组 220 人开始时间: 2014年7月11日最近更新: 2018年8月20日

研究概览

暂无简介。

•1. Completion of study treatment in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-020-DMD).
•2. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
•3. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 6-week follow-up period.
•4. Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 220
•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

•1. Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], magnesium stearate).
•2. Ongoing participation in any other therapeutic clinical trial.
•3. Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect
•the safety of the patient, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

发起方