Clinical Outcomes of Early Kasai Surgery With Umbilical Cord MSCs in Biliary Atresia

NCT07011199 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: No.16373/EC/KEPK-RSDK/2025
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial with historical control is to evaluate whether umbilical cord-derived mesenchymal stem cell (UC-MSC) therapy can improve clinical outcomes in infants with biliary atresia undergoing Kasai surgery before 90 days of age. The main questions it aims to answer are: Does UC-MSC therapy improve liver function parameters (bilirubin, albumin, liver enzymes, coagulation profile)? Does UC-MSC therapy reduce complications such as anemia, ascites, jaundice, and improve PELD scores? Does UC-MSC therapy improve overall survival compared to standard Kasai surgery alone? Researchers will compare the group receiving UC-MSC in 2025-2027 with a historical control group of patients who previously underwent Kasai surgery without UC-MSC therapy. Participants will: Undergo preoperative evaluation, including laboratory and imaging tests. Receive Kasai surgery combined with intraoperative trans-portal vein injection of 20 million UC-MSCs. Be monitored postoperatively through serial laboratory tests, imaging (Fibroscan), and clinical assessments at scheduled intervals. Be followed up for potential serious adverse events and survival outcomes.

Conditions

Biliary Atresia; Kasai Portoenterostomy; Mesenchymal Stem Cell Transplantation; Liver Function Disorders; Liver Fibrosis; Survival Rate

Outcome Measures

Primary Outcomes (9)

• Serum Albumin Level

  Measurement of serum albumin as marker of liver synthetic function

  Baseline (Pre-op), 4 weeks, 6 weeks, 12 weeks, 24 weeks, and 12 months post-operation

• Total Bilirubin Level

  Measurement of bilirubin levels (total, direct, indirect)

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Gamma Glutamyl Transferase (GGT)

  Measurement of liver enzyme levels

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Alanine Aminotransferase (ALT)

  Liver injury marker

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Aspartate Aminotransferase (AST)

  Liver injury marker

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Prothrombin Time (PT), aPTT, INR

  Coagulation parameters

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Alpha Fetoprotein (AFP)

  Tumor marker

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Alkaline Phosphatase (ALP)

  Liver and bone function marker

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Lactate Dehydrogenase (LDH)

  Tissue injury marker

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

Secondary Outcomes (5)

• Anemia Status

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Ascites

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Jaundice Status

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Pediatric End-Stage Liver Disease (PELD) Score

  Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months

• Overall Survival (OS)

  From the surgery date up to the end of the study (12 months maximum follow-up)

Study Arms (2)

Intervention Group (UC-MSC Group)

EXPERIMENTAL

Participants in this group are infants diagnosed with biliary atresia who undergo Kasai portoenterostomy before 90 days of age. During the operation, they receive intraoperative trans-portal vein injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) at a dose of 20 million cells diluted in 5 mL autologous plasma.

Biological: Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs); Procedure: Kasai Portoenterostomy

Control Group (Historical Control Group)

PLACEBO COMPARATOR

Participants in this group are historical patients who previously underwent Kasai portoenterostomy before 90 days of age at the same institution but did not receive UC-MSC therapy. Data for this group is collected retrospectively.

Procedure: Kasai Portoenterostomy

Interventions

Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) BIOLOGICAL

Intraoperative trans-portal vein injection of 20 million mesenchymal stem cells derived from umbilical cord tissue, suspended in 5 mL autologous plasma.

Intervention Group (UC-MSC Group)

Kasai Portoenterostomy PROCEDURE

Surgical procedure to create a portoenterostomy for biliary drainage in infants with biliary atresia.

Control Group (Historical Control Group); Intervention Group (UC-MSC Group)

Eligibility Criteria

• Age: 0 Days - 90 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Infants diagnosed with biliary atresia confirmed by intraoperative findings.
• Underwent Kasai portoenterostomy at an age less than 90 days.
• Parents or legal guardians have provided informed consent to participate in the study and follow all study procedures.

You may not qualify if:

• Infants with severe malnutrition.
• Infants with major congenital anomalies other than biliary atresia.
• Infants with positive tumor markers.

Sponsors & Collaborators

• Universitas Diponegoro: lead

Study Sites (1)

Dr. Kariadi General Hospital Medical Center

Semarang, Central Java, 50244, Indonesia

Location

MeSH Terms

Conditions

Biliary Atresia; Liver Cirrhosis

Interventions

Portoenterostomy, Hepatic

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

Intervention Hierarchy (Ancestors)

Anastomosis, Surgical; Surgical Procedures, Operative; Biliary Tract Surgical Procedures; Digestive System Surgical Procedures

Study Officials

• Avriana Pety Wardani, MD, Sp.BA

  Dr. Kariadi General Hospital Medical Center

  PRINCIPAL INVESTIGATOR

• Erik Prabowo, Dr, MD, M.Si.Med, Sp.B-KBD

  Dr. Kariadi General Hospital Medical Center

  STUDY DIRECTOR

• Kevin Christian Tjandra, MD

  Universitas Diponegoro

  STUDY CHAIR

• Danendra Rakha Putra Respati, MD

  Universitas Diponegoro

  STUDY CHAIR

Central Study Contacts

Avriana Pety Wardani, MD, Sp. BA

CONTACT

+62 81229515791; avrianagarg@gmail.com

Kevin Christian Tjandra, MD

CONTACT

+62 87700771775; kevinchristian2841@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Sp. BA

Study Record Dates

• First Submitted: May 30, 2025
• First Posted: June 8, 2025
• Study Start: June 17, 2025
• Primary Completion: April 17, 2026
• Study Completion: April 17, 2026
• Last Updated: June 8, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

ID (1)