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
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study
研究概览
简要总结
The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
详细描述
This is a two-arm, randomized, double-blind and double-dummy, placebo controlled study to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with TSC. The study consists of 3 phases for each patient: screening, core blinded phase, and open-label follow-up phase. Patients who meet the eligibility criteria will be randomized to receive vigabatrin or rapamycin. The randomization ratio is 1:1. Randomization will be stratified by the sex and the presence of epileptiform activity on baseline videoEEG (video electroencephalography) recording (yes versus no). Approximately 60 infants are planned to be enrolled in the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Double (Participant, Investigator)
盲法说明
Triple
入排标准
- 年龄范围
- 4 Weeks 至 16 Weeks（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged from 4 up to 16 weeks (44-56 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
排除标准
- •history of seizures prior to randomization,
- •history of antiepileptic treatment,
- •history of treatment with mTOR (mammalian Target of Rapamycin) inhibitor,
- •gestational age below 44 weeks at the day of randomization,
- •body weight lower than 3 kg at the day of randomization,
- •SEGA (Subependymal Giant Cell Astrocytoma) 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 1 month 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.
研究组 & 干预措施
Vigabatrin arm
Vigabatrin in capsules co-administered with placebo in liquid.
干预措施: Vigabatrin (Drug)
Vigabatrin arm
Vigabatrin in capsules co-administered with placebo in liquid.
干预措施: Placebo (Drug)
Rapamycin arm
Rapamycin in liquid co-administered with placebo in capsules.
干预措施: Rapamycin (Drug)
Rapamycin arm
Rapamycin in liquid co-administered with placebo in capsules.
干预措施: Placebo (Drug)
结局指标
主要结局
Summarized volume of TSC-associated tumors ≥ 125% of initial value within the blinded phase of the study
时间窗: 730 days
Occurrence of clinical seizures in the blinded phase of the study,
时间窗: 730 days
次要结局
- The risk of drug-resistant epilepsy at any point of the study(730 days)
- Parameters of physical development (weight gain history) across the whole study(730 days)
- The risk for high risk of autism assessed with psychological test at 6, 12, 18, 24 months(6, 12, 18, 24 months)
- Occurrence of adverse events within the blinded phase of the study(730 days)
- Parameters of physical development (height gain history) across the whole study(730 days)
- Number of adverse events across the whole study(730 days)
- Total volume of TSC-associated tumors within the blinded phase and the whole study(730 days)
- The risk for low developmental quotient (< 70 points in Bayley Scales of Infant Development, measured at the end of the blinded phase and at the end of the entire study) at the end of the study(730 days)
研究者
Katarzyna Kotulska
Head of the Department of Neurology and Epileptology at The Children's Memorial Health Institute
Children's Memorial Health Institute, Poland