Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis, a Randomized Double-blind Placebo Controlled Trial
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 140
- 试验地点
- 1
- 主要终点
- Change of alkaline phosphatase (ALP)
研究概览
简要总结
Ursodeoxycholic acid is the mainstay treatment medicine for primary biliary cholangitis(PBC). About 1/3 of the patients do not respond to UDCA, which is defined as refractory PBC. Mesenchymal stem cells (MSC) has been reported to improve the outcomes of PBC patients. Randomization controlled studies are needed to confirm the long term effect of MSC treatment for refractory PBC. This study aimed to investigate the safety and efficacy of mesenchymal stem cells in PBC patients that do not respond to UDCA treatment. This study is an double-blind multicenter randomized and placebo-controlled study. Patients with with refractory PBC will be randomly assigned to receive MSC treatment plus UDCA or UDCA alone (control). Three times of MSC infusion (0.1-1x10E6 cells/kg body weight) via peripheral vein will be given to the experimental group (once in 4 weeks). The primary outcome is absolute change in alkaline phosphatase. Secondary outcomes are changes of other liver function indices such as ALT TBIL AST GGT, improve of symptoms and liver histology.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Randomization will be blinded to participant, care provider, investigator and outcome assessors.
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
- 否
入选标准
- •informed consent
- •Age between 18-70 years old
- •BMI between 17-28
- •Meeting at least 2 of the 3 criteria: (1) positive for anti-mitochondial antibody (AMA)；(2) Elevated ALP or GGT；(3) PBC diagnosis confirmed by liver biopsy
- •Serum ALP >/=1.67 ULN after at leat 6 months treatment with UDCA 1(3-17mg/kg/day) -
排除标准
- •Pregnancy, breast-feeding females
- •Use of liver-toxic drugs over 2 week within 6 months prior to recruitment；
- •refractory variceal bleeding, uncontrolled hepatoencephalopathy or uncontrolled ascites.
- •Acute of chronic kidney failure.
- •Commodities with other liver diseases such as viral hepatitis, alcoholic liver disease, auto-immune hepatitis or liver cancer
- •Severe cardiovascular disease；
- •liver transplantation listed patients；
- •ALT/AST over 5xULN，or total bilirubin >85umol/l
- •anticipated need for liver transplantation within 1 year according to mayo risk score
- •Other candidates who are judged to be not applicable to this study by doctors
研究组 & 干预措施
MSC group
mesenchymal stem cell transplantation via peripheral vein: 0.1-1x10E6 MSCs/kg body weight administered via peripheral vein at week 0, 4, 8 plus UDCA.
干预措施: mesenchymal stem cell transplantation or placebo infusion via peripheral vein (Procedure)
control
placebo infusions (placebo infusion differs from the experimental infusion in only that placebo has no mesenchymal stem cells) will be administered via peripheral vein at week 0, 4, 8 plus UDCA.
干预措施: mesenchymal stem cell transplantation or placebo infusion via peripheral vein (Procedure)
结局指标
主要结局
Change of alkaline phosphatase (ALP)
时间窗: 1 year
The absolute value change of ALP after 1 year of the initial stem cell treatment
次要结局
- Change of other liver function indices(1 year)
- Change of liver histology(1 year)
研究者
Han Ying
Prof. Dr.
Xijing Hospital of Digestive Diseases