Clinical study on PTC743 in Patients with Leigh's Encephalomyelopathy (Continued Dosing Study)

Penematsa Vinay0 个研究点目标入组 12 人开始时间: 2020年12月3日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•1) Patients diagnosed with Leigh's encephalomyelopathy who completed the preceding study and who expressed the desire to continue the treatment with this drug
•2) Patients judged by the principal investigator or sub-investigator to show no problems in safety during the preceding study and for whom the treatment may be continued
•3) Patients for whom the legal representative was given an adequate explanation of the objectives and details of this study, and the anticipated drug efficacy, pharmacological action, and risks, for whom the understanding of the legal representative was obtained, and for whom the legal representative provided voluntary informed consent in writing; Also, patients from whom voluntary written assent was obtained to the extent that is possible.

•1) Patients with a previous history of allergies to PTC743, sesame oil or sesame
•2) Patients with a previous history of allergies to Vitamin E
•3) Patients showing a tendency for bleeding at the time point of Visit 9 (Month 6) or showing abnormal values for the prothrombin time (PT) at Visit 9, international normalized ratio (INR), or activated partial thromboplastin time (APTT) and judged by the principal investigator or sub-investigator to be inappropriate for inclusion in this study
•4) Patients being treated with an anti-coagulant drug
•5) Any other patients judged to be inappropriate for inclusion as a subject in this study by the principal investigator or a sub-investigator

发起方

Penematsa Vinay