Primary Biliary CirrhosisPhase 1/2UCMSCBANCT04522869
Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Biliary Atresia
A Phase 1/2 study testing Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation in people with Primary Biliary Cirrhosis, run by Vinmec Research Institute of Stem Cell and Gene Technology at 2 sites in Vietnam.
Trial summary
Biliary atresia (BA) is the most frequent cause of chronic cholestasis in neonates, accounting for at least 50% of pediatric liver transplantation. BA incidence is estimated to range from 1:5000 to 1:19000 live births. All patients will die due to complications of liver cirrhosis if the operation is not performed. Read more about this study
- Who is it for?
- Ages 5 months to 2 years
- Conditions the study is for
- Primary Biliary Cirrhosis
- What is being tested?
- Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesVietnam
- Study dates
- 10 Aug 2019to 25 Oct 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 5 months to 2 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children diagnosed with liver cirrhosis due to biliary atresia after Kasai's operation
- From 5 months to 2 years old
- Weight ≥ 6 kg
- Patients with manifestation of cirrhosis on liver biopsy during Kasai's operation.
- Parents or primary caregivers signed the informed consent form.
Who cannot join
- Under 6kg or over 2 years old
- Coagulation disorders
- Allergy to anesthetic agents
- Active infections
Trial locations and contacts
2 sites in 1 country.
Vietnam2 sites
Vinmec International Hospital Times City
Hanoi, Hanoi, 100000
Vinmec Research Institute of Stem cell and Gene Technology
Hanoi, 10000
About this study
Official title: Evaluation Safety and Efficacy of Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia: A Matched Control Prospective Study
Biliary atresia (BA) is the most frequent cause of chronic cholestasis in neonates, accounting for at least 50% of pediatric liver transplantation. BA incidence is estimated to range from 1:5000 to 1:19000 live births. All patients will die due to complications of liver cirrhosis if the operation is not performed. Recently, mesenchymal stem cell (MSC) transplantation has been found as a promising therapy for liver cirrhosis in adults. Bone marrow-derived stem cell transplantation was also performed successfully for children with BA. Compared to MSC isolation from bone marrow, isolating MSCs from umbilical cord (UC) tissue is a less invasive procedure.
Furthermore, UC-derived MSCs (UC-MSCs) have been demonstrated to be safe and effective for liver cirrhosis in adults and different pediatric diseases, including liver cirrhosis due to primary biliary cirrhosis. The investigators will compare the outcomes of 17 Kasai operated BA patients who receive UC-MSC transplantation to 17 BA patients who only undergo Kasai operation. Two transplantations of UC - MSCs will be performed via the hepatic artery: the first transplant will be performed at baseline, and the second one will be performed 6 months later with a dosage of 1 million MSCs per kg of body weight. The frequency and severity of the adverse events or serious adverse events associated with UC-MSC injection at 72 hours post-injection will be used to assess the safety. The efficacy of the therapy will be measured using Pediatric End-Stage Liver Disease (PELD) score, liver function, and liver biopsy. This study would open a novel cell therapy to improve outcomes of patients with BA.
Read the detailed description
The study protocol was approved by the Vinmec International Hospital Ethics Committee, and National Ethics Committees. The stem cell products are conducted in accordance with (GMP) requirements and Good Clinical Practice (GCP). All patients and primary caregivers will receive a written consent form, a cover letter and a clear explanation of the safety issues, potential risks and benefits, and the procedure involved. Moreover, patients will be provided the updated results related to disease and the study during conducting the study and will be fully funded by the project.
- Conditions
- Primary Biliary Cirrhosis
- Being tested
- Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation
- Keywords
- liver cirrhosis, biliary atresia, Kasai's operation
- Medical subject headings
- Liver Cirrhosis, Biliary, Liver Cirrhosis, Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) TransplantationBiological
Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia
| Study group | Role | What they receive |
|---|---|---|
| Umbilical cord blood - derived mesenchymal stem cells | Experimental | 17 patients with BA underwent Kasai operation and then will received two doses of UC-MSCs at 1x106 cells/kg (body weight) administered via hepatic artery Receives: Biological: Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation |
| Control group | No intervention | 17 patients with BA will be conducted Kasai operation only |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change of PELD scores during study
The PELD score is calculated using the following formula:
PELD Score = 0.480 * ln (Bilirubin in mg/dL) + 1.857 * ln (INR) - 0.687 * ln (Albumin in g/dL) + 0.436 if the patient is <1 year old + 0.667 if there growth failure
Measured: baseline, 3 months, 6 months, 9 months, 12 months
The change of albumin (Liver function)
Levels of albumin (g/dL)
Measured: up to the 12-month period following treatment
The change of total bilirubin (Liver function)
Total bilirubin level (mg/dL)
Measured: up to the 12-month period following treatment
The change of prothrombin time
Prothrombin time (second)
Measured: up to the 12-month period following treatment
The change of liver biopsy
change of liver biopsy
Measured: up to the 12-month period following treatment
Secondary outcomes (1)
The number of Adverse Events (AE) and Serious Adverse Events (SAE)
Adverse Events (AE) and Serious Adverse Events (SAE) after MSC transplantation
Measured: baseline, 3 months, 6 months, 9 months, 12 months (time frame: up to the 12-month period following treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2020
- Study start10 Aug 2019
- Primary completion25 Sep 2021
- Study completion25 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Biliary CirrhosisStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene Technology
- Sponsor type
- University, hospital or other
- Collaborators
- Number 2 Children's Hospital, Ho Chi Minh City
- Responsible party
- Sponsor
- Investigators
- Liem Nguyen, Principal investigator, Vinmec Research Institute of Stem Cell and Gene Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04522869
- Sponsor's protocol ID
- ISC.19.10
- Acronym
- UCMSCBA
- Status last verified
- Jun 2024
- First submitted
- 4 Sep 2019
- Last updated
- 6 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: When the study complete
Publications
Nguyen TL, Nguyen HP, Bui TH, Phan TKT, Ngo DM, Ha TTH, Tran TT, Luu NAT, Phan TK, Ho PD. Outcomes of Allogeneic Umbilical Cord Mesenchymal Stem Cell Infusion for Liver Cirrhosis Due to Biliary Atresia After Kasai Operation. J Pediatr Surg. 2025 Nov;60(11):162624. doi: 10.1016/j.jpedsurg.2025.162624. Epub 2025 Sep 2.
PubMed 40907882
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation Safety and Efficacy of Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia: A Matched Control Prospective Study
- ClinicalTrials.gov ID
- NCT04522869
- Acronym / sponsor ID
- UCMSCBA / ISC.19.10
- Conditions
- Primary Biliary Cirrhosis
- Keywords
- liver cirrhosis, biliary atresia, Kasai's operation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene TechnologyUniversity, hospital or other
- Collaborators
- Number 2 Children's Hospital, Ho Chi Minh City
- Study start
- 10 Aug 2019 (actual)
- Primary completion
- 25 Sep 2021 (actual)
- Study completion
- 25 Oct 2021 (actual)
- First posted
- 21 Aug 2020 (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.
Biliary atresia (BA) is the most frequent cause of chronic cholestasis in neonates, accounting for at least 50% of pediatric liver transplantation. BA incidence is estimated to range from 1:5000 to 1:19000 live births. All patients will die due to complications of liver cirrhosis if the operation is not performed. Recently, mesenchymal stem cell (MSC) transplantation has been found as a promising therapy for liver cirrhosis in adults. Bone marrow-derived stem cell transplantation was also performed successfully for children with BA. Compared to MSC isolation from bone marrow, isolating MSCs from umbilical cord (UC) tissue is a less invasive procedure.
Furthermore, UC-derived MSCs (UC-MSCs) have been demonstrated to be safe and effective for liver cirrhosis in adults and different pediatric diseases, including liver cirrhosis due to primary biliary cirrhosis. The investigators will compare the outcomes of 17 Kasai operated BA patients who receive UC-MSC transplantation to 17 BA patients who only undergo Kasai operation. Two transplantations of UC - MSCs will be performed via the hepatic artery: the first transplant will be performed at baseline, and the second one will be performed 6 months later with a dosage of 1 million MSCs per kg of body weight. The frequency and severity of the adverse events or serious adverse events associated with UC-MSC injection at 72 hours post-injection will be used to assess the safety. The efficacy of the therapy will be measured using Pediatric End-Stage Liver Disease (PELD) score, liver function, and liver biopsy. This study would open a novel cell therapy to improve outcomes of patients with BA.
Detailed description
The study protocol was approved by the Vinmec International Hospital Ethics Committee, and National Ethics Committees. The stem cell products are conducted in accordance with (GMP) requirements and Good Clinical Practice (GCP). All patients and primary caregivers will receive a written consent form, a cover letter and a clear explanation of the safety issues, potential risks and benefits, and the procedure involved. Moreover, patients will be provided the updated results related to disease and the study during conducting the study and will be fully funded by the project.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Umbilical cord blood - derived mesenchymal stem cells | Experimental | Biological: Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation17 patients with BA underwent Kasai operation and then will received two doses of UC-MSCs at 1x106 cells/kg (body weight) administered via hepatic artery |
| Control group | No intervention | –17 patients with BA will be conducted Kasai operation only |
Who may qualify
- Ages
- 5 months to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Children diagnosed with liver cirrhosis due to biliary atresia after Kasai's operation
- From 5 months to 2 years old
- Weight ≥ 6 kg
- Patients with manifestation of cirrhosis on liver biopsy during Kasai's operation.
- Parents or primary caregivers signed the informed consent form.
Key exclusion criteria (4 listed)
- Under 6kg or over 2 years old
- Coagulation disorders
- Allergy to anesthetic agents
- Active infections
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 Cell (UC -MSC) TransplantationUmbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia | Biological | Umbilical cord blood - derived mesenchymal stem cells |
Primary outcome
The change of PELD scores during study
Time frame: baseline, 3 months, 6 months, 9 months, 12 months
The PELD score is calculated using the following formula: PELD Score = 0.480 * ln (Bilirubin in mg/dL) + 1.857 * ln (INR) - 0.687 * ln (Albumin in g/dL) + 0.436 if the patient is <1 year old + 0.667 if there growth failure
The change of albumin (Liver function)
Time frame: up to the 12-month period following treatment
Levels of albumin (g/dL)
The change of total bilirubin (Liver function)
Time frame: up to the 12-month period following treatment
Total bilirubin level (mg/dL)
The change of prothrombin time
Time frame: up to the 12-month period following treatment
Prothrombin time (second)
The change of liver biopsy
Time frame: up to the 12-month period following treatment
change of liver biopsy
1 secondary outcome
The number of Adverse Events (AE) and Serious Adverse Events (SAE)
Time frame: baseline, 3 months, 6 months, 9 months, 12 months (time frame: up to the 12-month period following treatment)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2024.
- Derived from this study: PubMed 40907882Nguyen TL, Nguyen HP, Bui TH, Phan TKT, Ngo DM, Ha TTH, Tran TT, Luu NAT, Phan TK, Ho PD. Outcomes of Allogeneic Umbilical Cord Mesenchymal Stem Cell Infusion for Liver Cirrhosis Due to Biliary Atresia After Kasai Operation. J Pediatr Surg. 2025 Nov;60(11):162624. doi: 10.1016/j.jpedsurg.2025.162624. Epub 2025 Sep 2.
- Individual participant dataSharing planned: No. When the study complete
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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. This study is not recruiting at the moment.
Liem Nguyen principal investigator
Vinmec Research Institute of Stem Cell and Gene Technology
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation in people with Primary Biliary Cirrhosis, run by Vinmec Research Institute of Stem Cell and Gene Technology at 2 sites in Vietnam.
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 5 inclusion and 4 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: finished, as of the sponsor's last update on 6 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Vietnam. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of PELD scores during study (time frame: baseline, 3 months, 6 months, 9 months, 12 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04522869, which also lists 1 linked publication.
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 6 Jun 2024.