Stem Cell Applications in Biliary Atresia Patients

NCT06564740 | Recruiting

• 1 other identifier: Biliar Atresia Stem Cell
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

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.

Conditions

Biliary Atresia; Fibrosis, Liver; Stem Cell

Keywords

biliary atresia; liver fibrosis; stem cell

Outcome Measures

Primary Outcomes (1)

• Adverse events

  Adverse events will be assessed during the stem cell applications, as well as at 1 week, 1 month, 3 months, 6 months, 1 year, and 2 years after the application.

  2 years

Secondary Outcomes (13)

• Pediatric End-Stage Liver Disease (PELD) score

  2 years

• ALT levels (Alanine transaminase)

  2 years

• AST levels (Aspartate trasnaminase)

  2 years

• Direct bilirubin levels

  2 years

• Total bilirubin levels

  2 years

Study Arms (2)

Stem Cell Application Group

EXPERIMENTAL

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.

Drug: Stem Cell

Control

SHAM COMPARATOR

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.

Other: Control

Interventions

Stem Cell 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.

Stem Cell Application Group

Control 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.

Control

Eligibility Criteria

• Age: 2 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Necmi Kadıoğlu Hospital: lead

Study Sites (1)

Esenyurt State Hospital

Istanbul, Istanbul, 34340, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Biliary Atresia; Liver Cirrhosis

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Digestive System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Liver Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Mustafa Azizoglu, MD, PhD

  Esenyurt State Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mustafa Azizoglu, MD, PhD

CONTACT

+905447448244; mdmazizoglu@gmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Doctor

Model Details: 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.

Study Record Dates

• First Submitted: August 17, 2024
• First Posted: August 21, 2024
• Study Start: June 1, 2024
• Primary Completion (Estimated): May 30, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: September 19, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)