Rapamycin as Treatment for ADPKD: The Role of Biomarkers in Predicting a Response to Therapy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- Total Kidney Volume (mL)
研究概览
简要总结
Currently the only approved use for rapamycin (sirolimus) is for immunosuppression after renal transplantation.
This trial is designed to determine whether rapamycin is safe and effective treatment for patients with polycystic kidney disease (ADPKD). Patients will be followed by volumetric magnetic resonance imaging (MRI) to observe for change in kidney (and cyst) size. Blood and urine samples will also be collected to evaluate for change in biomarkers with treatment.
详细描述
This is a 2 year, open label trial to evaluate the role of rapamycin as treatment for ADPKD. Patients will be randomized 2:1 to the rapamycin arm or to standard therapy. The dose of rapamycin will be adjusted so that patients obtain 24 trough levels of 4-6ng/ml. There will be a volumetric MRI measurement at the start and end of the treatment period. Patients will be monitored every 4 months throughout 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
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •adult ADPKD patients aged 18-70
- •combined kidney volume >1200 ml
- •estimated creatinine clearance >60 ml/min
- •absence of implanted ferromagnetic objects
排除标准
- •uncontrolled hyperlipidemia
- •Proteinuria >500 mg/day
- •unstable cerebral aneurysm
- •active coronary artery disease
- •diagnosis of another systemic condition affecting kidney function (ie: diabetes, hepatitis B or C, HIV)
- •diagnosis of cancer other than skin cancer
- •pregnancy or lactation
- •presence of implanted ferromagnetic objects
研究组 & 干预措施
Standard of Care-Placebo
Standard of Care
干预措施: Standard of Care-Placebo (Other)
Rapamycin
Drug: Rapamycin
Other Names:
sirolimus The starting dose of rapamycin will be 1 mg daily. The dose will be increased as needed to achieve a 24 hour trough level of 4-6 ng/ml.
干预措施: Rapamycin (Drug)
结局指标
主要结局
Total Kidney Volume (mL)
时间窗: 2 years
次要结局
- Glomerular Filtration Rate (Kidney Function)(2 years)