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NCT Number: NCT07797673

Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia

Biliary atresia is a progressive liver disease in infants where liver transplantation is often the only long-term option once cirrhosis develops. However, organ shortages, high costs, risks of graft rejection, and the need for lifelong immunosuppression make transplantation difficult for many families. This double-blind randomized clinical trial evaluates whether injecting umbilical cord-mesenchymal stem cells directly into the liver during Kasai portoenterostomy is safe and effective as an additional treatment. Umbilical cord stem cells have strong anti-inflammatory and anti-fibrotic properties, and they carry a low risk of immune rejection. Patients undergoing the Kasai procedure are randomly assigned to receive either direct intrahepatic stem cell injections or a placebo. Participants are followed for 180 days post-surgery to monitor safety, liver function, and changes in liver stiffness.

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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 Hepatic Insufficiency Kasai Operation Liver Cirrhosis Liver Diseases Liver Fibrosis Liver Function Failure Pathologic Processes Pathological Conditions, Signs and Symptoms Stem Cell

Age range

30 day–90 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Pediatric patients aged 30 to 90 days.
  • Suspected biliary atresia based on clinical evaluation and diagnostic workup.
  • Biliary atresia diagnosis confirmed by intraoperative cholangiography.
  • Undergoing Kasai portoenterostomy at Cipto Mangunkusumo Hospital.
  • Written informed consent provided by a parent or legally authorized representative.

Exclusion criteria

  • Presence of congenital heart disease.
  • Diagnosis of Down syndrome.
  • Diagnoses other than biliary atresia confirmed by intraoperative cholangiography (e.g., choledochal cyst)

Drop-out Criteria:

  • Subjects will be dropped from the study if they develop postoperative anastomotic leakage

Treatment and study plan

Umbilical Cord-Mesenchymal Stem Cells (UC-MSC)

Biological

Intraoperative direct intraparenchymal injections of umbilical cord-mesenchymal stem cells at a dose of 10^5 cells/kg in 0.5 mL of 0.9% normal saline, distributed across hepatic segments 3, 4, 5, and 6 in both liver lobes

Kasai portoenterostomy

Procedure

Standard surgical resection of extrahepatic biliary remnants with a Roux-en-Y portoenterostomy to restore bile drainage in biliary atresia patients

Primary outcomes

  1. Total Bilirubin Level

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Measurement of total bilirubin level

Secondary outcomes

  1. Direct Bilirubin Level

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Measurement of direct bilirubin level

  2. Indirect Bilirubin Level

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Measurement of indirect bilirubin levels

  3. AST (Aspartate Transaminase) Levels

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Hepatocellular injury marker

  4. ALT(Alanine Transaminase) Levels

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Hepatocellular injury marker

  5. Gamma-glutamyl Transferase (GGT)

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Cholestasis marker

  6. Serum Albumin Level

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Marker of liver synthesis function and nutrition status

  7. Prothrombin Time (PT), INR

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Coagulation parameter to assess liver synthesis function

  8. Complete Blood Count

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Hematology panel including hemoglobin, leukocyte count, platelet count, absolute neutrophil count (ANC), and hematocrit.

  9. Pediatric End-Stage Liver Disease-Creatinine (PELD-Cr) Score

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    The Pediatric End-Stage Liver Disease-Creatinine (PELD-Cr) score is used to assess liver disease severity and estimate mortality risk in pediatric patients with end-stage liver disease. In accordance with Organ Procurement and Transplantation Network (OPTN) policy, the minimum score for this scale is capped at 6 (any calculated laboratory score below 6 is reported as 6), and there is no upper limit or maximum score. Higher PELD-Cr scores indicate greater liver disease severity and a worse clinical prognosis.

  10. Jaundice Clearance

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Postoperative jaundice resolution, assessed through clinical evaluation and total/direct serum bilirubin levels.

  11. Liver Fibrosis Stages

    Time frame: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

    Evaluated by measuring liver stiffness via Acoustic Radiation Force Impulse (ARFI) ultrasound, mapped to the METAVIR Fibrosis Staging Scale. The scale ranges from a minimum score of 0 (F0: no fibrosis) to a maximum score of 4 (F4: cirrhosis). Higher scores indicate greater liver fibrosis severity and a worse clinical outcome.

  12. Regulatory T-cell (Treg) levels

    Time frame: Post-Operative Day 7

    Regulatory T-cell (Treg) levels as an immunological parameter, measured postoperatively using flow cytometry and reported in standard laboratory units.

  13. CD11c Levels

    Time frame: Post-Operative Day 7

    CD11c-positive immune cell marker levels, measured postoperatively via flow cytometry and reported in standard laboratory units.

  14. Postoperative Cholangitis Incidence

    Time frame: Baseline, POD 7, POD 30, POD 90, POD 180

    Incidence of postoperative cholangitis, diagnosed based on clinical symptoms, physical examination, and supporting diagnostic tests as evaluated by the attending physician.

  15. Length of Stay

    Time frame: From the date of surgery until initial hospital discharge or death during the same period of care with the surgery, whichever occurs first (assessed up to 12 months).

    Calculated as the number of consecutive days from the date of surgery to the date of initial hospital discharge or in-hospital death during the index hospitalization. Re-admissions following initial discharge are excluded.

  16. All-Cause Mortality

    Time frame: From the date of study enrollment until death, assessed up to 12 months.

    Evaluated as the incidence of death from any cause occurring during the study period. Cause of death are verified through electronic medical record documentation, official death certificates, or direct patient/family follow-up contact.

Study contacts

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

Irene Effendy, MD

CONTACT

[email protected]

+62 851 5891 6100

Tri Hening Rahayatri, MD, PhD

CONTACT

[email protected]

+62 816 1381 223

Sponsors and collaborators

Lead sponsor

Dr Cipto Mangunkusumo General Hospital

Other

Collaborators

  • PT. Kimia Farma (Persero) Tbk

Registry information

Official study title

Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia: A Double-blind Randomized Controlled Trial

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Sep 1, 2026
Registry last updated
Sep 1, 2026

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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