Phase 1/2 Study of UC-MSC Treatment for the Evaluation the Efficacy and Safety in Patients With Liver Cirrhosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 266
- 试验地点
- 1
- 主要终点
- survival time
研究概览
简要总结
Liver cirrhosis (LC) represents a late stage of progressive hepatic fibrosis characterized by distortion of the hepatic architecture and formation of regenerative nodules. The liver transplantation is one of the only effective therapies available to such patients. However, lack of donors, surgical complications, rejection, and high cost are it's serious problems. The potential for stem cells to differentiate into hepatocytes cells was recently confirmed. Particularly, autologous bone marrow-derived mesenchymal stem cell (BM-MSC) has been demonstrated to decrease MELD score and increase serum albumin in the patients with decompensated liver cirrhosis. Therefore, the investigators propose a hypothesis that umbilical cord-derived MSCs (UC-MSC) can also improve the disease conditions of LC patients, particularly reducing the decompensated conditions in these patients.
详细描述
Liver cirrhosis (LC) represents a late stage of progressive hepatic fibrosis characterized by distortion of the hepatic architecture and formation of regenerative nodules. The liver transplantation is one of the only effective therapies available to such patients. However, lack of donors, surgical complications, rejection, and high cost are it's serious problems.
The potential for stem cells to differentiate into hepatocytes cells was recently confirmed. In particular, bone marrow-derived mesenchymal stem cell (BM-MSC) transplantation has been applicated in the clinic for treat several human diseases such as GVHD, cardiac injury and brain injury, and displayed good tolerance and efficiency. BM-MSC has also been used to treat human liver diseases such as liver failure and liver cirrhosis. In a phase 1 study, autologous BM-MSC transplantation has potential to decrease MELD score and increase serum albumin in the patients with decompensated liver cirrhosis.
The purpose of this study is to learn whether and how umbilical cord-derived MSCs (UC-MSC) can improve the longer term survival in patients with liver cirrhosis. This study will also look at how well BM-MSC is tolerated and its safety in LC patients.
Participants in the study will be randomly assigned to one of two treatment arms:
Arm A: Participants will receive conserved treatment plus three times UC-MSC treatment at 4-week intervals.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Liver cirrhosis
- •Negative pregnancy test (female patients in fertile age)
- •written consent
排除标准
- •Hepatocellular carcinoma or other malignancies
- •Presence of significant extrahepatic biliary disease (e.g. CBD stone, PSC, etc.)
- •Cardiac, renal or respiratory failure
- •Active thrombosis of the portal or hepatic veins
研究组 & 干预措施
conventional plus MSC treatment
participants will receive conventional treatment plus a dose of MSC from day 0 through the week 8 study visit. Participants will then be followed until the 75 months study visit.
干预措施: conventional plus MSC treatment (Drug)
conventional plus placebo treatment
participants will receive conventional plus placebo treatment from day 0 through the week 8 study visit. Participants will then be followed until the 75 months study visit.
干预措施: conventional plus placebo treatment (Drug)
结局指标
主要结局
survival time
时间窗: 75 months
incidence of HCC events
时间窗: 75 months
次要结局
- The levels of serum albumin(48 weeks)
- The levels of serum total bilirubin(48 weeks)
- The levels of serum prothrombin activity(48 weeks)
- complications(48 weeks)
- the levels of serum cholinesterase(48 weeks)
研究者
Fu-Sheng Wang
the Institute of Translational hepatology
Beijing 302 Hospital