Stem Cell Applications in Biliary Atresia Patients

Официальное название

Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants with Biliary Atresia: a Prospective Randomized Controlled Trial

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат Stem Cell
  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.
• Другое 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 these patients will continue to be administered.

Первичные конечные точки

• Adverse events [Срок оценки: 2 years]

Вторичные конечные точки (12)

• Pediatric End-Stage Liver Disease (PELD) score [Срок оценки: 2 years]
• ALT levels (Alanine transaminase) [Срок оценки: 2 years]
• AST levels (Aspartate trasnaminase) [Срок оценки: 2 years]
• Direct bilirubin levels [Срок оценки: 2 years]
• Total bilirubin levels [Срок оценки: 2 years]
• GGT levels (Gama glutamil transferase) [Срок оценки: 2 years]
• Liver biopsy [Срок оценки: 2 years]
• Level of cirrhosis [Срок оценки: 2 years]
• Re-operation rate [Срок оценки: 2 years]
• Liver transplantation [Срок оценки: 2 years]
• Albumin levels [Срок оценки: 2 years]
• INR (international normalized ratio) [Срок оценки: 2 years]

Критерии участия

Критерии включения

• 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).

Критерии исключения

• Epilepsy
• Neurological disorders
• Coagulation disorders
• Diabetes
• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Простое слепое

Основная цель

Лечение

Центры проведения

Turkey (Türkiye) · 1 центр

• Esenyurt State Hospital — Istanbul

Идентификаторы

NCT: NCT06564740 · Biliar Atresia Stem Cell

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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