A phase 1 extension study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy (FCMD) (NS035-P1-OE)

未提供0 个研究点目标入组 12 人开始时间: 待定

适应症

试验速览

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•Patients with FCMD who completed the TKY-TWMU-2021 trial
•Patients whose legal guardian capable of providing informed consent has provided written informed consent upon thorough understanding of the study procedure. Efforts should be made so that the subjects themselves give voluntary assent after having been provided with an explanation according to their ability to understand.

•Patients in whom the principal investigator (or sub-investigator) determined that a safety-related adverse event occurred during the TKY-TWMU-2021 trial
•Patients who underwent treatment aimed at producing fukutin or fukutin-related proteins after completion of the TKY-TWMU-2021 trial
•Patients who are pregnant, or who cannot agree to use an appropriate contraceptive method from the first dose of the investigational drug until 6 months after the final dose(including cases where menarche or spermarche occurs during the clinical trial period)
•Patients with a history of epilepsy who have had status epilepticus within the past year since the consent was obtained.

发起方

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