A Phase I, Open-label Trial on the Safety and Efficacy of Human Umbilical Cord-derived Mesenchymal Stem Cell Administration in the Management of Adult Liver Cirrhosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Number of Adverse events and serious adverse events (AEs or SAEs)
研究概览
简要总结
This is a phase I, open label, single arm trial using UC-MSCs to treat patients with liver cirrhosis that includes 20 patients. The primary outcome measure will be change in MELD score at 3, 6, and 12 months after UC-MSC transplantation from baseline. The safety is assessed by frequency and severity of the adverse event or serious adverse event associated with stem cell injection. This study could reconfirm the efficacy of stem cell transplantation for liver cirrhosis and would open a novel cell therapy for the treatment of adult liver cirrhosis.
详细描述
Liver cirrhosis is defined as the histological development of regenerative nodules surrounded by fibrous bands and causes by many forms of liver diseases and conditions such as hepatitis and chronic alcoholism. Liver cirrhosis has become one of the major causes of morbidity and mortality with over one million people died due to cirrhosis in 2010 worldwide. Currently, the only curative treatment for end-stage liver disease is liver transplantation. However, the shortage in donor organ availability and the side-effect associated with long-term immunosuppression after transplant requires the development of new alternative therapies for liver cirrhosis. Recent clinical trials demonstrate that bone marrow-derived stem cells (BMDSCs) as well as umbilical cord-derive mesenchymal stem cells (UC-MSCs) improve liver function in patients with liver cirrhosis. In Vietnam, however, there are no clinical trials to date that investigate the potential of UC-MSCs for liver cirrhosis management. This is a phase I, open label, single arm trial using UC-MSCs to treat patients with liver cirrhosis that includes 20 patients. The primary outcome measure will be change in MELD score at 3, 6, and 12 months after UC-MSC transplantation from baseline. The safety is assessed by frequency and severity of the adverse event or serious adverse event associated with stem cell injection. This study could reconfirm the efficacy of stem cell transplantation for liver cirrhosis and would open a novel cell therapy for the treatment of adult liver cirrhosis
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 18 to 70 years old.
- •Patients diagnosed of liver cirrhosis: splenomegaly, elevated hepatic enzymes and/or esophageal varices detected by endoscopy, or liver biopsy demonstrated liver cirrhosis
- •Be willing to complete the study and sign the consent form.
排除标准
- •Younger than 18 or older than 70 years old
- •Recent infection
- •Patients with history of chronic diseases such as cancer, chronic hepatitis, chronic kidney disease.
- •Any clinically significant blood coagulation disorder(s)
结局指标
主要结局
Number of Adverse events and serious adverse events (AEs or SAEs)
时间窗: up to the 12-month period following the first UC-MSCs infusion
To assess safety: the number of AEs or SAEs during and after allogenic umbilical cord-mesenchymal stem cells (UC-MSCs) infusion
次要结局
- Change of liver function through the Model for End Stage Liver Disease (MELD) score(3 months, 6 months, 12 months after the UC-MSCs infusion)
- Change in health-related quality of life using Chronic Liver Disease Questionnaire - (CLDQ)(3 months, 6 months, 12 months after the UC-MSCs infusion)