Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).
详细描述
This was an open label, single arm, multi-center, Phase IV Post-Approval Commitment (PAC) study with once daily oral dose of 10 mg everolimus in participants with renal AML associated with TSC. There were three separate phases in this study: a Screening phase, an Open-label treatment phase where participants received everolimus for 48 weeks or until disease progression, and a Treatment discontinuation follow-up phase for patients who discontinue study drug for reasons other than disease progression. Every participant had an End of Treatment visit within 28 days after last dose and a safety follow-up visit 30 days after last dose.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Eligible for treatment with everolimus as per the locally approved label.
- •Presence of at least one AML ≥ 3 cm in its longest diameter using CT or MRI.
排除标准
- •AML related bleeding or embolization during the 6 months prior to enrollment.
- •History of myocardial infarction, angina or stroke related to atherosclerosis.
- •Impaired lung function.
- •Significant hematological or hepatic abnormality.
- •Any severe and/or uncontrolled medical conditions.
研究组 & 干预措施
Everolimus
Participants targeted to receive Everolimus tablets 10 mg orally once daily for 48 weeks.
干预措施: Everolimus (Drug)
结局指标
主要结局
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: From the start of treatment to 30 days after end of treatment, assessed up to maximum duration of 52 weeks
Percentage of participants with AEs and SAEs including significant changes from baseline in vital signs, electrocardiograms and laboratory parameters (laboratory assessments included hematology, biochemistry, coagulation and urinalysis) qualifying and reported as AEs. The percentage of participants in each category is reported in the table.
次要结局
- Percentage of Participants With Best Overall Response (BOR) Status of Angiomyolipoma (AML) Response During Maximum Treatment Duration of 48 Weeks(Baseline, 48 weeks)
- Percentage of Participants With Best Overall Response Status of Angiomyolipoma (AML) Progression During Maximum Treatment Duration of 48 Weeks(Baseline, 48 weeks)
- Percentage of Participants With Severe Renal Impairment(Baseline, 48 weeks)
- Percentage of Participants With NCI CTCA Grade 3/4 Serum Creatinine(Baseline, 48 weeks)