A Multicenter, Randomized, Placebo-controlled, Double-blind Study on the Efficacy, Safety and Tolerability of Everolimus in Preventing End-stage Renal Disease (ESRD) in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 431
- 试验地点
- 1
- 主要终点
- Primary Efficacy Analysis of Total Kidney Volume (mITT Set, Multiple Imputation)
研究概览
简要总结
This study will assess whether everolimus (RAD001) is effective in preventing cyst and kidney expansion as well as worsening of renal function in patients with ADPKD and whether the application of 5 mg/day everolimus as monotherapy is safe and well tolerated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Everolimus
Patients in the everolimus group initially received 5 mg/day everolimus divided in 2 equal doses (i.e. 2.5 mg b.i.d.). Dose adjustments were performed to achieve a blood trough level of 3-8 ng/mL (maximum daily dose: 10 mg/day [5 mg b.i.d.]).
干预措施: Everolimus (Drug)
Placebo
Placebo tablets equivalent to the dosage of everolimus 5 mg/day, divided in 2 equal doses.
干预措施: Placebo (Drug)
结局指标
主要结局
Primary Efficacy Analysis of Total Kidney Volume (mITT Set, Multiple Imputation)
时间窗: Baseline, Month 24
Everolimus (RAD001) compared to placebo with respect to the change from baseline in total kidney volume at Month 24.
次要结局
- Course of Calculated GFR (mL/Min/1.73 m^2) From Month 24 to Month 60(Months 24, 36, 48 and 60)
- Calculated GFR, Change From Baseline at Month 60 by Baseline cGFR(Months 24, 36, 48 and 60)
- Changes in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)(Baseline, Months 12 and 24)
- Calculated GFR (mL/Min/1.73 m^2), Change From Baseline by Visit(Months 3, 6, 9, 12, 18 and 24)