An Open-label, Prospective Clinical Trial to Evaluate the Effectiveness and Safety of Sirolimus to Reduce Cyst Growth in ADPKD Patients With Massive Polycystic Liver
试验速览
- 阶段
- 2 期
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Total liver volume
研究概览
简要总结
The purpose of this study is to evaluate the effectiveness and safety of Sirolimus in reducing liver volume in autosomal dominant polycystic kidney disease.
详细描述
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common causes of end stage renal disease (ESRD), affecting an estimated 0.2% of population. Of ADPKD patients, 58% in 15-24 year, 85% in 25-34 year, and 94% in 35-46 year olds suffer from polycystic liver in addition to polycystic kidneys. Several anti-proliferative drugs have been used in clinical trials to stop cyst growth both in liver and kidneys. Among them, octreotide and sirolimus have been shown to be one of the most promising drugs to reduce cyst volume. Sirolimus already has been used as one of the most potential oral immunosuppressants. Moreover, the serum trough level is quite easy to measure. Sirolimus is the mTOR inhibitor that has been proven to be effective in reducing cyst growth both in animal models. However, its efficacy and safety is not well proven in previous studies. This is a open-label, prospective study to evaluate the effectiveness and safety of Sirolimus to reduce cyst growth in ADPKD patients with massive polycystic liver.
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
- •Age 18 - 65
- •Patients diagnosed as ADPKD based upon the unified criteria for ultrasonographic diagnosis of ADPKD
- •Polycystic liver with total liver volume > 2500 mL or symptomatic polycystic liver
- •Estimated glomerular filtration rate (IDMS-traceable MDRD equation) >= 30 mL/min/1.73m2
排除标准
- •Concomitant systemic renal parenchymal or urinary tract disease (random urine albumin-to-creatinine ratio > 500 mg/g)
- •WBC < 4,000/uL, platelet < 100,000/uL, or hemoglobin < 10.0 g/dL
- •Diabetes mellitus, cancer, or psychiatric disorder
- •Increased liver enzymes (2-fold above normal value)
- •Hypercholesterolemia (fasting cholesterol > 200mg/dL) or hypertriglyceridemia (>150 mg/dL) not controlled by lipid lowering therapy
- •Infection with hepatitis B, C, HIV
- •Any condition that could prevent full comprehension of the purpose and risks of the study
- •Pregnant or lactating women or fertile women without effective contraception
- •History of intervention, such as cyst aspiration or embolization in past 1 year
研究组 & 干预措施
Sirolimus
Sirolimus administration group starting dose: 2mg/day target trough level: 4-10 ng/dL
干预措施: Sirolimus (Drug)
结局指标
主要结局
Total liver volume
时间窗: 12 months
Change in total liver volume
次要结局
- Total kidney volume(24 month)
- Estimated glomerular filtration rate(24 month)
- Urinary biomarker(24 month)
- Total liver volume(24 months)