NCT07797673

Brief Summary

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P50-P75 for phase_1

Timeline
26mo left

Started Sep 2026

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress4%
Sep 2026Dec 2028

First Submitted

Initial submission to the registry

August 23, 2026

Completed
9 days until next milestone

First Posted

Study publicly available on registry

September 1, 2026

Completed
Same day until next milestone

Study Start

First participant enrolled

September 1, 2026

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2028

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2028

Last Updated

September 9, 2026

Status Verified

September 1, 2026

Enrollment Period

2 years

First QC Date

August 23, 2026

Last Update Submit

September 4, 2026

Conditions

Biliary AtresiaKasai OperationLiver FibrosisStem CellLiver Function Failure

Keywords

Biliary AtresiaKasai portoenterostomyUmbilical cord mesenchymal stem cellUC-MSCLiver fibrosisRegenerative therapyDouble-blind randomized controlled trial

Outcome Measures

Primary Outcomes (1)

  • Total Bilirubin Level

    Measurement of total bilirubin level

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

Secondary Outcomes (16)

  • Direct Bilirubin Level

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

  • Indirect Bilirubin Level

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

  • AST (Aspartate Transaminase) Levels

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

  • ALT(Alanine Transaminase) Levels

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

  • Gamma-glutamyl Transferase (GGT)

    Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180

  • +11 more secondary outcomes

Study Arms (2)

UC-MSC Group

EXPERIMENTAL

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 Group

PLACEBO COMPARATOR

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

Umbilical Cord-Mesenchymal Stem Cells (UC-MSC)BIOLOGICAL

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

UC-MSC Group
Kasai PortoenterostomyPROCEDURE

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

Placebo GroupUC-MSC Group

Eligibility Criteria

Age30 Days - 90 Days
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cipto Mangunkusumo Hospital

Jakarta Pusat, DKI Jakarta, 10430, Indonesia

RECRUITING

MeSH Terms

Conditions

Biliary AtresiaLiver CirrhosisHepatic Insufficiency

Interventions

Portoenterostomy, Hepatic

Condition Hierarchy (Ancestors)

Bile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesDigestive System AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLiver DiseasesFibrosisPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Anastomosis, SurgicalSurgical Procedures, OperativeBiliary Tract Surgical ProceduresDigestive System Surgical Procedures

Study Officials

  • Radiana Dhewayani Antarianto, MD, M.Biomed, PhD

    University of Indonesia, Cipto Mangunkusumo Hospital

    PRINCIPAL INVESTIGATOR

  • Fatima Safira Alatas, MD, PhD

    University of Indonesia, Cipto Mangunkusumo Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Tri Hening Rahayatri, MD, PhD

CONTACT

+62 816 1381 223rahayatri@gmail.com

Irene Effendy, MD

CONTACT

+62 851 5891 6100databasebped@gmail.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Pediatric Surgeon, Head of Pediatric Surgery Division, MD, PhD, Principal Investigator

Study Record Dates

First Submitted

August 23, 2026

First Posted

September 1, 2026

Study Start

September 1, 2026

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

December 1, 2028

Last Updated

September 9, 2026

Record last verified: 2026-09

Data Sharing

IPD Sharing
Will not share

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.

Locations

ID(1)