Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants With Biliary Atresia: A Prospective Randomized Controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 64
- 试验地点
- 1
- 主要终点
- Adverse events
研究概览
简要总结
Recently, mesenchymal stem cell (MSC) transplantation has emerged as a promising treatment for liver cirrhosis in adults. Additionally, bone marrow-derived stem cell transplantation has shown success in treating children with biliary atresia (BA). This study aims to evaluate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA through a multicentric randomized controlled trial.
详细描述
Biliary atresia (BA) is the most common cause of chronic cholestasis in neonates and accounts for at least 50% of pediatric liver transplants. The incidence of BA is estimated to range from 1:5000 to 1:19000 live births. If the operation is not performed, all patients will die due to complications of liver cirrhosis. Recently, mesenchymal stem cell (MSC) transplantation has been found to be a promising treatment for liver cirrhosis in adults. Stem cell transplantation derived from bone marrow has also been successfully applied to children with BA. The aim of this study is to demonstrate the efficacy of Umbilical Cord-Derived Mesenchymal Stem Cell (UC-MSC) therapy in BA by planning a multicentric randomized controlled trial.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 2 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Infants were diagnosed with liver cirrhosis due to biliary atresia following Kasai's operation.
- •The patients two months old or older and exhibited signs of cirrhosis after the procedure, including hepatomegaly, congestive splenomegaly, elevated liver enzymes, esophageal varices (confirmed by endoscopy), and cirrhosis (confirmed by liver biopsy).
排除标准
- •Neurological disorders
- •Coagulation disorders
- •Syndromic type biliary atresia
- •Allergies to anesthetic agents
- •Severe health conditions such as cancer or failure of the heart, lungs, liver, or kidneys, active infections, and severe psychiatric disorders.
研究组 & 干预措施
Stem Cell Application Group
In this group, UC-MSC transplantation will be administered twice to each patient in the study group via the hepatic artery: the first transplantation will be performed post-surgery at the beginning, and the second one will be performed 6 months later, with a dose of 1 million UC-MSC/kg. The applications will be applied after kasai portoenterostomy.
干预措施: Stem Cell (Drug)
Control
In this group, UC-MSC will not be administered. This group serves as a passive control. The standard treatments that are routinely provided to Biliary Atresia patients will continue to be administered.
干预措施: Control (Other)
结局指标
主要结局
Adverse events
时间窗: 2 years
Adverse events will be assessed during the stem cell applications, as well as at 1 week, 1 month, 3 months, 6 months, 1 year, and 2 years after the application.
次要结局
- Pediatric End-Stage Liver Disease (PELD) score(2 years)
- ALT levels (Alanine transaminase)(2 years)
- AST levels (Aspartate trasnaminase)(2 years)
- Direct bilirubin levels(2 years)
- Total bilirubin levels(2 years)
- GGT levels (Gama glutamil transferase)(2 years)
- Liver biopsy(2 years)
- Level of cirrhosis(2 years)
- Re-operation rate(2 years)
- Liver transplantation(2 years)
- Albumin levels(2 years)
- INR (international normalized ratio)(2 years)
- Cholangitis(2 years)
研究者
Mustafa Azizoğlu
Medical Doctor
Necmi Kadıoğlu Hospital