Menu
Recruiting NCT06564740

Stem Cell Applications in Biliary Atresia Patients

No phase Interventional Biliary Atresia Fibrosis, Liver Stem Cell

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Stem Cell, Control.
Who it may be relevant to
Registry conditions: Biliary Atresia, Fibrosis, Liver, Stem Cell. Basic parameters: 2 months — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Turkey (Türkiye)
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Detailed description

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.

Interventions

  • Drug 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.
  • Other 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.

Primary outcome measures

  • Adverse events [Time frame: 2 years]
Secondary outcome measures (12)
  • Pediatric End-Stage Liver Disease (PELD) score [Time frame: 2 years]
  • ALT levels (Alanine transaminase) [Time frame: 2 years]
  • AST levels (Aspartate trasnaminase) [Time frame: 2 years]
  • Direct bilirubin levels [Time frame: 2 years]
  • Total bilirubin levels [Time frame: 2 years]
  • GGT levels (Gama glutamil transferase) [Time frame: 2 years]
  • Liver biopsy [Time frame: 2 years]
  • Level of cirrhosis [Time frame: 2 years]
  • Re-operation rate [Time frame: 2 years]
  • Liver transplantation [Time frame: 2 years]
  • Albumin levels [Time frame: 2 years]
  • INR (international normalized ratio) [Time frame: 2 years]

Eligibility criteria

Inclusion criteria

  • 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).

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Single blind
Primary purpose
Treatment

Study locations

Turkey (Türkiye) · 1 center
  • Esenyurt State Hospital — Istanbul

Identifiers

NCT: NCT06564740 · Biliar Atresia Stem Cell

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗