Biliary AtresiaPhase 1ABMNCBANCT05517317
Autologous BMNC Infusion for Liver Cirrhosis in Children With BA
A Phase 1 study testing Autologous BMMC transplantation in people with Biliary Atresia, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site in Vietnam.
Trial summary
To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. Read more about this study
- Who is it for?
- Ages 2 months to 15 years
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- Autologous BMMC transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteVietnam
- Study dates
- 1 Jan 2015to 30 May 2022
- Phase
- Phase 1
- 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 2 months to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
- Two months old or older
- Patients with a manifestation of cirrhosis after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)
Who cannot join
- Epilepsy
- Coagulation disorders
- Allergy to anesthetic agents
- Severe health conditions such as cancer, failure of heart, lung, liver or kidney
- Active infections
- Severe psychiatric disorders
Trial locations and contacts
1 site in 1 country.
Vietnam1 site
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, 100000
About this study
Official title: Autologous Bone Marrow Mononuclear Cell Infusion for Liver Cirrhosis in Children With Biliary Atresia
To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery.
Read the detailed description
Biliary atresia (BA) is a progressive fibro-obliterative cholangiopathy and a fatal disease. Without surgery, children with BA rarely survive beyond three years of age. The reported prevalence of BA ranges from 1 in 9640 to 1 in 19,500 live births. In the past, most children with a "non-correctable" type of BA died without adequate treatment. Recently, stem cell administration has been applied in adults with liver cirrhosis and has shown promising outcomes. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery. Serum bilirubin, albumin, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, and prothrombin time were monitored at baseline, three months, six months and 12 months after the transplantation. Esophagoscopies and liver biopsies were performed in patients whose parents provided consent. This study aimed to evaluate both safety and hepatic function after BMMNC administration in these children.
- Conditions
- Biliary Atresia
- Being tested
- Autologous BMMC transplantation
- Medical subject headings
- Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous BMMC transplantationCombination product
One administration of autologous bone marrow mononuclear cells via the hepatic artery
| Study group | Role | What they receive |
|---|---|---|
| Autologous BMMC infusion | Experimental | One administration of autologous bone marrow mononuclear cells via the hepatic artery Receives: Combination Product: Autologous BMMC transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events and serious adverse events
To assess safety, the number of AEs or SAEs during stem cell administration (72 h) at 1 month, 3 months, 6 months, and 9 months after discharge will be evaluated
Measured: up to the 12-month period following treatment
Secondary outcomes (6)
The changes in cholestasis
Using Total Bilirubin (units: mg/dL) to measure the changes in cholestasis
Measured: up to the 12-month period following treatment
The changes in Liver function using Aspart transaminase
Using AST (Aspart transaminase) (units: U/L) to measure the changes in liver function.
Measured: up to the 12-month period following treatment
The changes in Liver function using Alanine transaminase
Using ALT (Alanine transaminase) (units: U/L) to measure the changes in liver function.
Measured: up to the 12-month period following treatment
The changes in Liver function using Gamma GT
Using GGT (Gamma GT) (units: U/L) to measure the changes in liver function.
Measured: up to the 12-month period following treatment
The changes in level of cirrhosis
Using PELD score (according to the suggestion of The Liver and Intestinal Organ Transplantation Committee in 2009). PELD is calculated based on three indicators: albumin (g/dL), bilirubin (units: mg/dL) and INR (international normalized ratio). Formula: PELD = 10 * (0.48 * ln(Serum Bilirubin) + 1.857 * ln(INR) - 0.687 * ln(Albumin) + (0.436 if patient is less than 1 year old) + (0.667 if patient has growth failure).
Evaluate the result:
If PELD <10: good results
If 10 <PELD <15: average results
If PELD> 15: bad results
Measured: up to the 12-month period following treatment
The changes in liver biopsy
Liver biopsy is a powerful clinical tool to evaluate the changes in the level of cirrhosis.
Measured: 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 posted26 Aug 2022
- Study start1 Jan 2015
- Primary completion30 May 2022
- Study completion30 May 2022
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 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
- Not applicable
- Intervention model
- Single group
- Model description
- Autologous BMMC infusion
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene Technology
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Liem Thanh Nguyen, PhD, Principal investigator, Vinmec Research Institute of Stem Cell and Gene Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05517317
- Sponsor's protocol ID
- ISC_22_88
- Acronym
- ABMNCBA
- Status last verified
- Aug 2022
- First submitted
- 22 Aug 2022
- Last updated
- 26 Aug 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Thanh LN, Nguyen HP, Kieu TPT, Duy MN, Ha HTT, Thi HB, Nguyen TQ, Pham HD, Tran TD. Modified Kasai operation combined with autologous bone marrow mononuclear cell infusion for biliary atresia. BMC Surg. 2024 Nov 20;24(1):368. doi: 10.1186/s12893-024-02669-9.
PubMed 39568009
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Bone Marrow Mononuclear Cell Infusion for Liver Cirrhosis in Children With Biliary Atresia
- ClinicalTrials.gov ID
- NCT05517317
- Acronym / sponsor ID
- ABMNCBA / ISC_22_88
- Conditions
- Biliary Atresia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene TechnologyUniversity, hospital or other
- Study start
- 1 Jan 2015 (actual)
- Primary completion
- 30 May 2022 (actual)
- Study completion
- 30 May 2022 (actual)
- First posted
- 26 Aug 2022 (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.
To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery.
Detailed description
Biliary atresia (BA) is a progressive fibro-obliterative cholangiopathy and a fatal disease. Without surgery, children with BA rarely survive beyond three years of age. The reported prevalence of BA ranges from 1 in 9640 to 1 in 19,500 live births. In the past, most children with a "non-correctable" type of BA died without adequate treatment. Recently, stem cell administration has been applied in adults with liver cirrhosis and has shown promising outcomes. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery. Serum bilirubin, albumin, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, and prothrombin time were monitored at baseline, three months, six months and 12 months after the transplantation. Esophagoscopies and liver biopsies were performed in patients whose parents provided consent. This study aimed to evaluate both safety and hepatic function after BMMNC administration in these children.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Autologous BMMC infusion
| Arm | Type | Receives |
|---|---|---|
| Autologous BMMC infusion | Experimental | Combination Product: Autologous BMMC transplantationOne administration of autologous bone marrow mononuclear cells via the hepatic artery |
Who may qualify
- Ages
- 2 months to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
- Two months old or older
- Patients with a manifestation of cirrhosis after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)
Key exclusion criteria (6 listed)
- Epilepsy
- Coagulation disorders
- Allergy to anesthetic agents
- Severe health conditions such as cancer, failure of heart, lung, liver or kidney
- 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 |
|---|---|---|
| Autologous BMMC transplantationOne administration of autologous bone marrow mononuclear cells via the hepatic artery | Combination product | Autologous BMMC infusion |
Primary outcome
Adverse events and serious adverse events
Time frame: up to the 12-month period following treatment
To assess safety, the number of AEs or SAEs during stem cell administration (72 h) at 1 month, 3 months, 6 months, and 9 months after discharge will be evaluated
6 secondary outcomes
The changes in cholestasis
Time frame: up to the 12-month period following treatment
The changes in Liver function using Aspart transaminase
Time frame: up to the 12-month period following treatment
The changes in Liver function using Alanine transaminase
Time frame: up to the 12-month period following treatment
The changes in Liver function using Gamma GT
Time frame: up to the 12-month period following treatment
The changes in level of cirrhosis
Time frame: up to the 12-month period following treatment
The changes in liver biopsy
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 26 Aug 2022.
- Derived from this study: PubMed 39568009Thanh LN, Nguyen HP, Kieu TPT, Duy MN, Ha HTT, Thi HB, Nguyen TQ, Pham HD, Tran TD. Modified Kasai operation combined with autologous bone marrow mononuclear cell infusion for biliary atresia. BMC Surg. 2024 Nov 20;24(1):368. doi: 10.1186/s12893-024-02669-9.
- Individual participant dataSharing planned: No
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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 Thanh Nguyen, PhD principal investigator
Vinmec Research Institute of Stem Cell and Gene Technology
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 Autologous BMMC transplantation in people with Biliary Atresia, run by Vinmec Research Institute of Stem Cell and Gene Technology at 1 site 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 3 inclusion and 6 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 26 Aug 2022. 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 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 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?
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?
Adverse events and serious adverse events (time frame: up to the 12-month period following treatment). 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 NCT05517317, 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 26 Aug 2022; AzTrials last refreshed its copy on 8 Nov 2026.