Pilot Study of Fenofibrate for Primary Biliary Cirrhosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Serum Level of Alkaline Phosphatase
研究概览
简要总结
This is a pilot study to evaluate the safety and efficacy of fenofibrate on patients with primary biliary cirrhosis who have an incomplete response to ursodeoxycholic acid.
详细描述
This is a multicenter open label pilot study to evaluate the efficacy of fenofibrate in 20 patients with PBC treated for 1 year. A randomized design would not be feasible at this stage of the research. Two sites are enrolling patients: University of Florida, Gainesville, and Mayo Clinic Rochester, with the first as the coordinating site. The total number of patients to start treatment will not exceed 20. Our endpoints are 1) surrogate markers of disease activity -serum alkaline phosphatase and immunoglobulin M; 2) a well validated prognostic index -Mayo risk score and 3) the NIDDK quality of life questionnaire.
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
- 21 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established diagnosis of PBC by serological (positive antimitochondrial antibodies) and/or histological evidence (diagnostic liver biopsy)
- •Previous treatment with UDCA 13-15 mg/kg/day for at least 1 year
- •Persistent elevation of serum alkaline phosphatase ≥ 2 times the upper limit of normal on two separate measurements
- •Female patients of childbearing age should have a pregnancy test done within -days of the beginning of this trial, and should agree to be on adequate contraception throughout the study period
- •Signed informed consent after careful review of the information and study details by one of the investigators
- •Exclusion Criteria
- •Hypersensitivity to fenofibrate
- •Prisoners and institutionalized subjects
- •Pregnant or nursing women
- •Anticipated need for liver transplantation in one year (estimated one year survival < 80%) as determined by the Mayo risk score. The Mayo risk score takes into account the patient's age, serum bilirubin, albumin and prothrombin time, as well as presence or absence of peripheral edema.
- •Recipients of liver transplantation
- •Recurrent variceal hemorrhage, uncontrolled encephalopathy or refractory ascites
- •Co-existing liver diseases, such as primary sclerosing cholangitis, acute or chronic hepatitis, alcoholic liver disease, choledocholithiasis, autoimmune hepatitis, cholangiocarcinoma
- •Acute or chronic renal failure
- •Known history of cholecystitis with intact gallbladder
- •Current use of statins, as the concomitant use of fibrates and statins would increase the risk of toxicity
排除标准
- 未提供
研究组 & 干预措施
Patients treated with Fenofibrate
Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle) 160 mg table per day for 1 year
干预措施: Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle) (Drug)
结局指标
主要结局
Serum Level of Alkaline Phosphatase
时间窗: 1 year
We analyzed whether there was a difference in median ALP at 1 year compared to baseline values.
次要结局
未报告次要终点