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NCT Number: NCT06564740

Stem Cell Applications in Biliary Atresia Patients

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.

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Key information

Conditions

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

Age range

2 month–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Esenyurt State Hospital

Istanbul, 34340, Turkey (Türkiye)

Location status: Recruiting

Location contact

Mustafa Azizoglu, MD, PhD

CONTACT

[email protected]

+905447448244

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

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.

Primary outcomes

  1. Adverse events

    Time frame: 2 years

    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.

Secondary outcomes

  1. Pediatric End-Stage Liver Disease (PELD) score

    Time frame: 2 years

    Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 * (0.48 * ln(Serum Bilirubin) + 1.857 * ln(INR) - 0.687 * ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result:

    If PELD <10: good results If 10 <PELD <15: average results If PELD> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).

  2. ALT levels (Alanine transaminase)

    Time frame: 2 years

    The levels of ALT will be closely monitored in both groups. The values for this parameter will be recorded and evaluated individually for each patient.

  3. AST levels (Aspartate trasnaminase)

    Time frame: 2 years

    The levels of AST will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  4. Direct bilirubin levels

    Time frame: 2 years

    The levels of direct bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  5. Total bilirubin levels

    Time frame: 2 years

    The levels of total bilirubin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  6. GGT levels (Gama glutamil transferase)

    Time frame: 2 years

    The levels of GGT will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  7. Liver biopsy

    Time frame: 2 years

    Liver biopsy is a crucial clinical tool for assessing the progression and severity of cirrhosis

  8. Level of cirrhosis

    Time frame: 2 years

    Level of cirrhosis will be assesed with PELD score. Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio). Formula: PELD = 10 * (0.48 * ln(Serum Bilirubin) + 1.857 * ln(INR) - 0.687 * ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure)). Evaluate the result:

    If PELD <10: good results If 10 <PELD <15: average results If PELD> 15: bad results Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).

  9. Re-operation rate

    Time frame: 2 years

    The patients who need further surgical intervention will be noted.

  10. Liver transplantation

    Time frame: 2 years

    Liver transplantation will be evaluated. The number of patients requiring a liver transplant, along with the timing of the need, will be recorded.

  11. Albumin levels

    Time frame: 2 years

    The levels of albumin will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  12. INR (international normalized ratio)

    Time frame: 2 years

    The levels of INR (international normalized ratio) will be closely monitored in both groups. The values for each parameter will be recorded and evaluated individually for each patient.

  13. Cholangitis

    Time frame: 2 years

    The number of cholangitis for each cases will be closely monitored in both groups. The frequency of cholangitis experienced by each patient will be recorded and evaluated.

Study contacts

Contact information is provided by the study sponsor or research team.

Mustafa Azizoglu, MD, PhD

CONTACT

[email protected]

+905447448244

Sponsors and collaborators

Lead sponsor

Necmi Kadıoğlu Hospital

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 21, 2024
Registry last updated
Sep 19, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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