Clinical trial NCT06564740: Stem Cell Applications in Biliary Atresia Patients
Official title: Umbilical Cord Derived Mesenchymal Stem Cell (UC-MSC) Transplantation in Infants with Biliary Atresia: a Prospective Randomized Controlled Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Biliary Atresia
Fibrosis, Liver
Stem Cell
Keywords: biliary atresia
liver fibrosis
stem cell
Lead sponsor: Necmi Kadıoğlu Hospital
Sponsor class: OTHER
First posted: 2024-08-21
Enrollment: 64
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This study is designed as a multicentric randomized controlled trial (RCT). Patients will be randomly divided into two groups. Treatment Group: 30 Kasai-operated BA patients who will receive UC-MSC transplantation, and Control Group: 30 BA patients who will only undergo Kasai operation. 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.
Primary purpose: TREATMENT

Brief summary: 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.

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.

Primary outcomes:
measure: Adverse events
timeFrame: 2 years
Secondary outcomes:
measure: Pediatric End-Stage Liver Disease (PELD) score
timeFrame: 2 years
measure: ALT levels (Alanine transaminase)
timeFrame: 2 years
measure: AST levels (Aspartate trasnaminase)
timeFrame: 2 years
measure: Direct bilirubin levels
timeFrame: 2 years
measure: Total bilirubin levels
timeFrame: 2 years
measure: GGT levels (Gama glutamil transferase)
timeFrame: 2 years
measure: Liver biopsy
timeFrame: 2 years
measure: Level of cirrhosis
timeFrame: 2 years
measure: Re-operation rate
timeFrame: 2 years
measure: Liver transplantation
timeFrame: 2 years
measure: Albumin levels
timeFrame: 2 years
measure: INR (international normalized ratio)
timeFrame: 2 years
measure: Cholangitis
timeFrame: 2 years
Other outcomes:
Locations:
city: Istanbul
country: Turkey
facility: Esenyurt State Hospital
Study officials:
affiliation: Esenyurt State Hospital
name: Mustafa Azizoglu, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: