Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia

Browse trials for

• Biliary Atresia 77
• Liver Cirrhosis 1,298
• Hepatic Insufficiency 113

Keywords

• Biliary Atresia
• Kasai portoenterostomy
• Umbilical cord mesenchymal stem cell
• UC-MSC
• Liver fibrosis
• Regenerative therapy
• Double-blind randomized controlled trial

Biliary Atresia

77 studies on the registry are indexed under Biliary Atresia; 25 are open to participants now.

This study's planned enrollment of 40 is below the median of 45 across 43 interventional studies indexed under Biliary Atresia.

Lead sponsor

Dr Cipto Mangunkusumo General Hospital is the lead sponsor of 39 studies on the registry; 7 are open to participants now.

Study arms

• Experimental
  UC-MSC Group

  Infants aged 30-90 days with intraoperative cholangiography-confirmed biliary atresia undergoing Kasai portoenterostomy receive intraoperative intraparenchymal injections of umbilical cord-derived mesenchymal stem cells (UC-MSCs) across hepatic segments 3, 4, 5, and 6, at a target dose of 10\^5 cells/kg (prepared in a 1 mL syringe at a concentration of 10\^5 cells/0.1 mL in 0.9% NaCl)

  Biological: Umbilical Cord-Mesenchymal Stem Cells (UC-MSC) · Procedure: Kasai Portoenterostomy

• Placebo comparator
  Placebo Group

  Infants aged 30-90 days with intraoperative cholangiography-confirmed biliary atresia undergoing Kasai portoenterostomy receive intraoperative intraparenchymal injections of a placebo solution (prepared in a 1 mL syringe containing 0.9% normal saline) across hepatic segments 3, 4, 5, and 6.

  Procedure: Kasai Portoenterostomy

Interventions

• BiologicalUmbilical Cord-Mesenchymal Stem Cells (UC-MSC)

  Intraoperative intraparenchymal injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) at a dose of 10\^5cells/kg, administered via a 1 mL syringe containing UC-MSCs at a concentration of 10\^5 cells/0.1mL in 0.9% NaCl, distributed across hepatic segments 3, 4, 5, and 6 in both liver lobes

• ProcedureKasai Portoenterostomy

  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

   Measurement of total bilirubin level

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

Secondary outcomes

1. Direct Bilirubin Level

   Measurement of direct bilirubin level

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

2. Indirect Bilirubin Level

   Measurement of indirect bilirubin levels

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

3. AST (Aspartate Transaminase) Levels

   Hepatocellular injury marker

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

4. ALT(Alanine Transaminase) Levels

   Hepatocellular injury marker

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

5. Gamma-glutamyl Transferase (GGT)

   Cholestasis marker

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

6. Serum Albumin Level

   Marker of liver synthesis function and nutrition status

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

7. Prothrombin Time (PT), INR

   Coagulation parameter to assess liver synthesis function

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

8. Complete Blood Count

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

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

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

   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.

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

10. Jaundice Clearance

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

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

11. Liver Fibrosis Stages

    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.

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

12. Regulatory T-cell (Treg) levels

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

    Time frame: Post-Operative Day 7

13. CD11c Levels

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

    Time frame: Post-Operative Day 7

14. Postoperative Cholangitis Incidence

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

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

15. Length of Stay

    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.

    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).

16. All-Cause Mortality

    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.

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

Individual participant data

Plan to share: No — There is no plan to make individual participant data (IPD) available. To protect pediatric patient privacy and maintain ethical confidentiality, raw participant data will not be shared. This restriction aligns with institutional policies and local data protection regulations.