An Open-label, Multi-center Long-term Safety Roll-over Study in Patients With Tuberous Sclerosis Complex (TSC) and Refractory Seizures Who Are Judged by the Investigator to Benefit From Continued Treatment With Everolimus After Completion of Study CRAD001M2304.
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 206
- 试验地点
- 71
- 主要终点
- Occurances of adverse events and serious adverse events
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be benefiting from continued treatment as judged by the investigator at the completion of EXIST-3
详细描述
This is a multi-center, open label study to evaluate the long term safety of everolimus in patients with TSC and refractory seizures currently being treated in the Novartis-sponsored study EXIST-3 study and are judged by their parent study investigator as benefiting from the current study treatment. The study is expected to remain open for approximately 10 years (or until 31-Dec-2022 in the UK) from First Patient First Visit (FPFV).
Patients will continue to be treated in this study until they are no longer benefiting from their everolimus treatment as judged by the Investigator or until one of the protocol defined discontinuation criteria is met or everolimus becomes commercially available according to local regulations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient is currently enrolled in the Novartis-sponsored EXIST-3 study, receiving everolimus, and has fulfilled all its requirements
- •Patient is currently benefiting from treatment with everolimus, as determined by the Investigator.
- •Patient has demonstrated compliance, as assessed by the Investigator,with the parent study protocol requirements.
- •Patient is willing and able to comply with scheduled visits and treatment plans.
- •Written informed consent/adolescent assent obtained prior to enrolling into the roll-over study.
排除标准
- •Patient has been permanently discontinued from everolimus study treatment in EXIST-3 study
- •Everolimus is approved for patients with TSC and refractory seizures and is reimbursed in the local country.
- •Patients who are receiving everolimus in combination with unapproved or experimental treatments for seizure control Anti-epileptic drug (AEDs) are allowed for the purpose of seizure control.
研究组 & 干预措施
everolimus
everolimus, 2mg dispersible tablets
干预措施: everolimus (Drug)
结局指标
主要结局
Occurances of adverse events and serious adverse events
时间窗: Day 1 up to approximately 10 years
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
次要结局
- Percentage of patients with clinical benefit(Day 1 up to approximately 10 years, assessed every 12 weeks,)