Stem Cell Applications in Biliary Atresia Patients

NCT ID: NCT06564740

Last Updated: 2024-09-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

64 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-01

Study Completion Date

2026-06-01

Brief Summary

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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

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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

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Biliary Atresia Fibrosis, Liver Stem Cell

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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Stem Cell Application Group

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.

Group Type EXPERIMENTAL

Stem Cell

Intervention Type DRUG

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.

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 Biliary Atresia patients will continue to be administered.

Group Type SHAM_COMPARATOR

Control

Intervention Type OTHER

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.

Interventions

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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.

Intervention Type DRUG

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.

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

2 Months

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Necmi Kadıoğlu Hospital

OTHER

Sponsor Role lead

Responsible Party

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Mustafa Azizoğlu

Medical Doctor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Mustafa Azizoglu, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Esenyurt State Hospital

Locations

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Esenyurt State Hospital

Istanbul, Istanbul, Turkey (Türkiye)

Site Status RECRUITING

Countries

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Turkey (Türkiye)

Central Contacts

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Mustafa Azizoglu, MD, PhD

Role: CONTACT

+905447448244

Facility Contacts

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Mustafa Azizoglu, MD, PhD

Role: primary

+905447448244

Other Identifiers

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Biliar Atresia Stem Cell

Identifier Type: -

Identifier Source: org_study_id

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