Biliary AtresiaPhase 1NCT07011199
Clinical Outcomes of Early Kasai Surgery With Umbilical Cord MSCs in Biliary Atresia
A Phase 1 study testing Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) and Kasai Portoenterostomy in people with Biliary Atresia and Kasai Portoenterostomy, run by Universitas Diponegoro at 1 site in Indonesia.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 to 90 days
- Conditions the study is for
- Biliary Atresia, Kasai Portoenterostomy, Mesenchymal Stem Cell Transplantation
- What is being tested?
- Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) 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
- 14 participants
- Where
- 1 siteIndonesia
- Study dates
- 17 Jun 2025to 17 Apr 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 0 to 90 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- Infants with severe malnutrition.
- Infants with major congenital anomalies other than biliary atresia.
- Infants with positive tumor markers.
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
Dr. Kariadi General Hospital Medical Center
Semarang, Central Java, 50244
Avriana Pety Wardani, MD, Sp.BA, +62 81229515791, avrianagarg@gmail.com
Avriana Pety Wardani, MD, Sp.BA
About this study
Official title: Evaluation of Clinical Outcomes in Biliary Atresia Patients Undergoing Kasai Surgery Before 90 Days of Age With Umbilical Cord-Derived Mesenchymal Stem Cell Therapy
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:
Read more
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
- Being tested
- Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs), Kasai Portoenterostomy
- Medical subject headings
- Biliary Atresia, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
Kasai PortoenterostomyProcedure or surgery
Surgical procedure to create a portoenterostomy for biliary drainage in infants with biliary atresia.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Procedure: Kasai Portoenterostomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum Albumin Level
Measurement of serum albumin as marker of liver synthetic function
Measured: 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)
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Gamma Glutamyl Transferase (GGT)
Measurement of liver enzyme levels
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Alanine Aminotransferase (ALT)
Liver injury marker
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Aspartate Aminotransferase (AST)
Liver injury marker
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Prothrombin Time (PT), aPTT, INR
Coagulation parameters
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Alpha Fetoprotein (AFP)
Tumor marker
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Alkaline Phosphatase (ALP)
Liver and bone function marker
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Lactate Dehydrogenase (LDH)
Tissue injury marker
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Secondary outcomes (5)
Anemia Status
Clinical and laboratory evaluation of anemia
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Ascites
Clinical evaluation via physical exam and ultrasound
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Jaundice Status
Clinical and laboratory bilirubin assessment
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Pediatric End-Stage Liver Disease (PELD) Score
Disease severity scoring system
Measured: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Overall Survival (OS)
Survival rate since intervention
Measured: From the surgery date up to the end of the study (12 months maximum follow-up)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2025
- Study start17 Jun 2025 (estimated)
- Primary completion17 Apr 2026 (estimated)
- Study completion17 Apr 2026 (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
- Crossover
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitas Diponegoro
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Avriana Pety Wardani, Dr. Kariadi General Hospital Medical Center
- Investigators
- Avriana Pety Wardani, MD, Sp.BA, Principal investigator, Dr. Kariadi General Hospital Medical CenterErik Prabowo, Dr, MD, M.Si.Med, Sp.B-KBD, Study director, Dr. Kariadi General Hospital Medical CenterKevin Christian Tjandra, MD, Study chair, Universitas DiponegoroDanendra Rakha Putra Respati, MD, Study chair, Universitas Diponegoro
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07011199
- Sponsor's protocol ID
- No.16373/EC/KEPK-RSDK/2025
- Status last verified
- May 2025
- First submitted
- 30 May 2025
- Last updated
- 8 Jun 2025
- 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 IPD available. The individual participant data will not be shared due to privacy concerns and ethical considerations related to the protection of identifiable health information of pediatric patients. Data sharing is also restricted by institutional policy and local regulations.
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Clinical Outcomes in Biliary Atresia Patients Undergoing Kasai Surgery Before 90 Days of Age With Umbilical Cord-Derived Mesenchymal Stem Cell Therapy
- ClinicalTrials.gov ID
- NCT07011199
- Acronym / sponsor ID
- No.16373/EC/KEPK-RSDK/2025
- Conditions
- Biliary Atresia, Kasai Portoenterostomy, Mesenchymal Stem Cell Transplantation, Liver Function Disorders, Liver Fibrosis, Survival Rate
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Universitas DiponegoroUniversity, hospital or other
- Study start
- 17 Jun 2025 (estimated)
- Primary completion
- 17 Apr 2026 (estimated)
- Study completion
- 17 Apr 2026 (estimated)
- First posted
- 8 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention Group (UC-MSC Group) | Experimental | Biological: Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs), Procedure: Kasai PortoenterostomyParticipants 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. |
| Control Group (Historical Control Group) | Placebo comparator | Procedure: Kasai PortoenterostomyParticipants 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. |
Who may qualify
- Ages
- 0 to 90 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- Infants with severe malnutrition.
- Infants with major congenital anomalies other than biliary atresia.
- Infants with positive tumor markers.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs)Intraoperative trans-portal vein injection of 20 million mesenchymal stem cells derived from umbilical cord tissue, suspended in 5 mL autologous plasma. | Biological | Intervention Group (UC-MSC Group) |
| Kasai PortoenterostomySurgical procedure to create a portoenterostomy for biliary drainage in infants with biliary atresia. | Procedure or surgery | Control Group (Historical Control Group), Intervention Group (UC-MSC Group) |
Primary outcome
Serum Albumin Level
Time frame: Baseline (Pre-op), 4 weeks, 6 weeks, 12 weeks, 24 weeks, and 12 months post-operation
Measurement of serum albumin as marker of liver synthetic function
Total Bilirubin Level
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Measurement of bilirubin levels (total, direct, indirect)
Gamma Glutamyl Transferase (GGT)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Measurement of liver enzyme levels
Alanine Aminotransferase (ALT)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver injury marker
Aspartate Aminotransferase (AST)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver injury marker
Prothrombin Time (PT), aPTT, INR
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Coagulation parameters
Alpha Fetoprotein (AFP)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Tumor marker
Alkaline Phosphatase (ALP)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Liver and bone function marker
Lactate Dehydrogenase (LDH)
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Tissue injury marker
5 secondary outcomes
Anemia Status
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Ascites
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Jaundice Status
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Pediatric End-Stage Liver Disease (PELD) Score
Time frame: Baseline, 4 weeks, 6 weeks, 12 weeks, 24 weeks, 12 months
Overall Survival (OS)
Time frame: From the surgery date up to the end of the study (12 months maximum follow-up)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2025.
- Individual participant dataSharing planned: No. There is no plan to make IPD available. The individual participant data will not be shared due to privacy concerns and ethical considerations related to the protection of identifiable health information of pediatric patients. Data sharing is also restricted by institutional policy and local regulations.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Avriana Pety Wardani, MD, Sp.BA principal investigator
Dr. Kariadi General Hospital Medical Center
Erik Prabowo, Dr, MD, M.Si.Med, Sp.B-KBD study director
Dr. Kariadi General Hospital Medical Center
Kevin Christian Tjandra, MD study chair
Universitas Diponegoro
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Umbilical Cord-Derived Mesenchymal Stem Cells (UC-MSCs) and Kasai Portoenterostomy in people with Biliary Atresia and Kasai Portoenterostomy, run by Universitas Diponegoro at 1 site in Indonesia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Indonesia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Avriana Pety Wardani, MD, Sp. BA (+62 81229515791, avrianagarg@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serum Albumin Level (time frame: Baseline (Pre-op), 4 weeks, 6 weeks, 12 weeks, 24 weeks, and 12 months post-operation). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2025.