Phase IV, Prospective Single Arm Study of Safety and Efficacy of Votubia (Everolimus) in Taiwanese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 4
- 试验地点
- 3
- 主要终点
- Percentage of participants with adverse events (AEs), Serious AEs (SAEs) and AEs of special interest (AESI)
研究概览
简要总结
The purpose of this prospective study is to assess the safety and efficacy of everolimus in Taiwanese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC) . Only patients who fulfil the local reimbursement criteria of everolimus for TSC-AML will be included in this study.
详细描述
This open-label, prospective, single-arm, multicenter Phase IV post approval commitment (PAC) study is planned to be conducted in approximately 10 patients with confirmed diagnosis of TSC-AML and who fulfil the local reimbursement criteria of everolimus for TSC-AML treatment.
The study will have a 30-day screening phase, and each patient will be on treatment up to 52 weeks. Enrollment will end at the latest within 52 weeks from Day 1 of the study, regardless of the number of patients actually recruited. After completion of the treatment phase/end of treatment (EOT), eligible patients will enter a 4-week safety follow up (FU) phase. Patients who continue to be on treatment beyond 52 weeks, based on the investigator's judgment will not be included in the 4-week safety FU phase.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult male or female patients from ≥ 18 years of age.
- •Signed informed consent must be obtained prior to participation in the study.
- •Participants with TSC associated with renal AML which is eligible for treatment with everolimus per local reimbursement criteria.
排除标准
- •Patients with severe hepatic impairment (Child-Pugh class C)
- •Any severe and/or uncontrolled medical conditions.
- •Pregnant or breast-feeding females.
- •Patients with hypersensitivity to the active substance, to other rapamycin derivatives, or to any of the excipients.
研究组 & 干预措施
Everolimus
Participants with confirmed diagnosis of TSC-AML and who fulfil the local (Taiwan) reimbursement criteria of everolimus for TSC-AML treatment
干预措施: Everolimus (Drug)
结局指标
主要结局
Percentage of participants with adverse events (AEs), Serious AEs (SAEs) and AEs of special interest (AESI)
时间窗: From first dose of study treatment up to 56 weeks
Percentage of participants with AEs, SAEs and AESIs.
次要结局
- Angiomyolipoma (AML) response rate(Up to 52 weeks)
- Percentage of participants with laboratory abnormalities(From screening up to 56 weeks)
- AML progression rate(Up to 52 weeks)