Biliary AtresiaPhase 1NCT07797673
Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia
A Phase 1 study testing Umbilical Cord-Mesenchymal Stem Cells (UC-MSC) and Kasai Portoenterostomy in people with Biliary Atresia and Kasai Operation, run by Dr Cipto Mangunkusumo General Hospital at 1 site in Indonesia.
Trial 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. Read more about this study
- Who is it for?
- Ages 30 to 90 days
- Conditions the study is for
- Biliary Atresia, Kasai Operation, Liver Fibrosis
- What is being tested?
- Umbilical Cord-Mesenchymal Stem Cells (UC-MSC) and Kasai Portoenterostomy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteIndonesia
- Study dates
- 1 Sep 2026to Dec 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 90 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Indonesia1 site, 1 recruiting
- Recruiting
Cipto Mangunkusumo Hospital
Jakarta Pusat, DKI Jakarta, 10430
Tri Hening Rahayatri, MD, PhD, +62 816 1381 223, rahayatri@gmail.com
Irene Effendy, MD, +62 851 5891 6100, databasebped@gmail.com
About this study
Official title: Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia: A Double-blind Randomized Controlled Trial
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.
- Being tested
- Umbilical Cord-Mesenchymal Stem Cells (UC-MSC), Kasai Portoenterostomy
- Keywords
- Biliary Atresia, Kasai portoenterostomy, Umbilical cord mesenchymal stem cell, UC-MSC, Liver fibrosis, Regenerative therapy, Double-blind randomized controlled trial
- Medical subject headings
- Biliary Atresia, Liver Cirrhosis, Hepatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
Kasai PortoenterostomyProcedure or surgery
Standard surgical resection of extrahepatic biliary remnants with a Roux-en-Y portoenterostomy to restore bile drainage in biliary atresia patients
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: 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. Receives: Procedure: Kasai Portoenterostomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Bilirubin Level
Measurement of total bilirubin level
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Secondary outcomes (16)
Direct Bilirubin Level
Measurement of direct bilirubin level
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Indirect Bilirubin Level
Measurement of indirect bilirubin levels
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
AST (Aspartate Transaminase) Levels
Hepatocellular injury marker
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
ALT(Alanine Transaminase) Levels
Hepatocellular injury marker
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Gamma-glutamyl Transferase (GGT)
Cholestasis marker
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Serum Albumin Level
Marker of liver synthesis function and nutrition status
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Prothrombin Time (PT), INR
Coagulation parameter to assess liver synthesis function
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Complete Blood Count
Hematology panel including hemoglobin, leukocyte count, platelet count, absolute neutrophil count (ANC), and hematocrit.
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
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.
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
Jaundice Clearance
Postoperative jaundice resolution, assessed through clinical evaluation and total/direct serum bilirubin levels.
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
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.
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
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.
Measured: Post-Operative Day 7
CD11c Levels
CD11c-positive immune cell marker levels, measured postoperatively via flow cytometry and reported in standard laboratory units.
Measured: Post-Operative Day 7
Postoperative Cholangitis Incidence
Incidence of postoperative cholangitis, diagnosed based on clinical symptoms, physical examination, and supporting diagnostic tests as evaluated by the attending physician.
Measured: Baseline, Post-Operative Day 7, Post-Operative Day 30, Post-Operative Day 90, Post-Operative Day 180
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.
Measured: 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).
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.
Measured: From the date of study enrollment until death, assessed up to 12 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2026
- Study start1 Sep 2026
- Primary completionSep 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary AtresiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dr Cipto Mangunkusumo General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- PT. Kimia Farma (Persero) Tbk
- Responsible party
- Principal investigator, Tri Hening Rahayatri, Dr Cipto Mangunkusumo General Hospital
- Investigators
- Radiana Dhewayani Antarianto, MD, M.Biomed, PhD, Principal investigator, University of Indonesia, Cipto Mangunkusumo HospitalFatima Safira Alatas, MD, PhD, Principal investigator, University of Indonesia, Cipto Mangunkusumo Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07797673
- Sponsor's protocol ID
- 26-06-0853
- Other IDs
- No.551/SK/R/UI/2026
- Status last verified
- Sep 2026
- First submitted
- 23 Aug 2026
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.