Randomized, placebo-controlled, double-blind and double-dummy clinical trial comparing the safety, tolerability, and efficacy of vigabatrin and rapamycin in a preventive treatment of infants with Tuberous Sclerosis Complex (ViRap) - ViRap

The Children's Memorial Health Institute0 个研究点目标入组 60 人开始时间: 2020年10月9日最近更新: 2023年11月13日

适应症

试验速览

暂无简介。

•For the inclusion in the study, patients must fulfil all the following inclusion criteria:
•-Male or female aged from 4 up to 16 weeks (41-56 weeks of gestational age) at the day of randomization
•-Parents/caregivers are willing to and able to give informed consent form for the participation in the study
•-Parents/caregivers are willing to and able to comply with all study requirements
•-Definite diagnosis of TSC according to the Consensus criteria (Northrup,2013)
•-At least 1 focus of cortical dysplasia disclosed on brain MRI
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 60
•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

•Patient may not enter the study if any of the following exclusion criteria are met:
•-history of seizures prior to randomization
•-history of antiepileptic treatment
•-history of treatment with mTOR inhibitor,
•-gestational age below 41 weeks at the day of randomization
•-body weight lower than 3 kg at the day of randomization
•-SEGA or other TSC- associated lesion requiring urgent surgical intervention
•-recent surgery within 1 month prior to the randomization
•-intercurrent infection at the date of randomization
•-known history of HIV seropositivity
•-live vaccination within 4 weeks prior to randomization
•-lack of first TBC and hepatitis B vaccinations
•-Any significant clinical, laboratory , ECG or other abnormalities, comorbidity or concomitant treatment which, in the opinion of the investigator, may either put a patient at significant risk associated with the participation in the study or may influence the results of the study.
•-Use of an investigational drug within 1 month prior to randomization

发起方

The Children's Memorial Health Institute