Stem Cell Applications in Biliary Atresia Patients

Necmi Kadıoğlu Hospital

Status: Enrolling

Conditions: Fibrosis, Liver; Stem Cell; Biliary Atresia

Treatments: Other: Control; Drug: Stem Cell

Study type: Interventional

Funder types: Other

Identifiers

NCT06564740

Biliar Atresia Stem Cell

Details and patient eligibility

About

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.

Full 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.

Enrollment: 64 estimated patients

Sex: All

Ages: 2 months to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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64 participants in 2 patient groups

Stem Cell Application Group

Experimental group

Description:

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.

Treatment:

Drug: Stem Cell

Control

Sham Comparator group

Description:

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.

Treatment:

Other: Control