NCT06564740

Stem Cell Applications in Biliary Atresia Patients

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

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 64 (estimated)
• Sponsor: Necmi Kadıoğlu Hospital · Academic / Other
• Sex: All
• Age: 2 Months – 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Biliary Atresia
• Fibrosis, Liver
• Stem Cell

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2024-06-01
• Primary completion: 2026-05-30
• Completion: 2026-06-01
• First posted: 2024-08-21
• Last updated: 2024-09-19

Locations

1 site across 1 country: Turkey (Türkiye)