Liver Cirrhosis, BiliaryPhase 2NCT03468699
Autologous Bone Marrow Mononuclear Stem Cell for Children Suffering From Liver Cirrhosis Due to Biliary Atresia
A Phase 2 study testing Autologous BMMC transplantation in people with Liver Cirrhosis, Biliary, run by Vinmec Research Institute of Stem Cell and Gene Technology at 2 sites in Vietnam.
Trial summary
The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation for Children Suffering from Liver Cirrhosis Due to Biliary Atresia Read more about this study
- Who is it for?
- Ages 1 year to 15 yearsHealthy volunteers can take part
- Conditions the study is for
- Liver Cirrhosis, Biliary
- What is being tested?
- Autologous BMMC transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 2 sitesVietnam
- Study dates
- 2 Jan 2017to 31 Oct 2021
- Phase
- Phase 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 1 year to 15 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Children were 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 after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)
Who cannot join
- Under 1 year old patients
- 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
2 sites in 1 country.
Vietnam2 sites
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, 100000
Vinmec Research Institute of Stem Cell and Gene Technology
Hanoi, 10000
About this study
Official title: Outcomes of Autologous Bone Marrow Mononuclear Stem Cell (BMMC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia After Kasai's Operation: An Open Label Uncontrolled Clinical Trial
The aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation for Children Suffering from Liver Cirrhosis Due to Biliary Atresia
Read the detailed description
This study is ferformed to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in the management of Liver Cirrhosis Due to Biliary Atresia after Kasai's operation of 20 patients at Vinmec Research Institute of Stem Cell and Gene Technology, Hanoi, Vietnam from January 2017 to December 2018
- Conditions
- Liver Cirrhosis, Biliary
- Being tested
- Autologous BMMC transplantation
- Keywords
- Autologous bone marrow Transplantation, biliary atresia, Kasai's operation, liver cirrhosis
- Medical subject headings
- Liver Cirrhosis, Biliary, Biliary Atresia, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous BMMC transplantationCombination product
Stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| Autologous BMMC transplantation | Experimental | Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward Receives: Combination Product: Autologous BMMC transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the changes in cholestasis
Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis
Measured: Baseline, 3, 6 months and 12 months after transplantation
Assess the changes in Liver function
Using AST (Aspart transaminase) and ALT (Alanin transaminase) (units: U/L), GGT (Gamma GT) (units: UI/L), and Bilirubin (units: mg/dL) to measure the changes in Liver function
Measured: Baseline, 3, 6 months and 12 months after transplantation
Assess 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
Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Measured: Baseline, 3, 6 months and 12 months after transplantation
Assess the changes in liver biopsy
Liver biopsy, in combination with history and physical examination data, is a powerful clinical tool for diagnosing and treating liver disease to evaluate the changes in liver biopsy
Measured: Baseline, 3, 6 months and 12 months after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2018
- Study start2 Jan 2017
- Primary completion30 Sep 2021
- Study completion31 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Liver Cirrhosis, BiliaryStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 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 Nguyen, Principal investigator, Vinmec Research Institute of Stem Cell and Gene Technology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03468699
- Sponsor's protocol ID
- VinmecRISCGT78
- Status last verified
- Jun 2024
- First submitted
- 26 Jan 2018
- Last updated
- 4 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Nicolas C, Wang Y, Luebke-Wheeler J, Nyberg SL. Stem Cell Therapies for Treatment of Liver Disease. Biomedicines. 2016 Jan 6;4(1):2. doi: 10.3390/biomedicines4010002.
PubMed 28536370Levicar N, Dimarakis I, Flores C, Tracey J, Gordon MY, Habib NA. Stem cells as a treatment for chronic liver disease and diabetes. Handb Exp Pharmacol. 2007;(180):243-62. doi: 10.1007/978-3-540-68976-8_11.
PubMed 17554512Irfan A, Ahmed I. Could Stem Cell Therapy be the Cure in Liver Cirrhosis? J Clin Exp Hepatol. 2015 Jun;5(2):142-6. doi: 10.1016/j.jceh.2014.03.042. Epub 2014 Mar 27.
PubMed 26155042Sharma S, Kumar L, Mohanty S, Kumar R, Datta Gupta S, Gupta DK. Bone marrow mononuclear stem cell infusion improves biochemical parameters and scintigraphy in infants with biliary atresia. Pediatr Surg Int. 2011 Jan;27(1):81-9. doi: 10.1007/s00383-010-2712-4.
PubMed 20857300Khan AA, Parveen N, Mahaboob VS, Rajendraprasad A, Ravindraprakash HR, Venkateswarlu J, Rao P, Pande G, Narusu ML, Khaja MN, Pramila R, Habeeb A, Habibullah CM. Management of hyperbilirubinemia in biliary atresia by hepatic progenitor cell transplantation through hepatic artery: a case report. Transplant Proc. 2008 May;40(4):1153-5. doi: 10.1016/j.transproceed.2008.03.110.
PubMed 18555137Nguyen TL, Nguyen HP, Ngo DM, Ha THT, Mai KA, Bui TH, Nguyen PV, Pham LH, Hoang DM, Cao ADT. Autologous bone marrow mononuclear cell infusion for liver cirrhosis after the Kasai operation in children with biliary atresia. Stem Cell Res Ther. 2022 Mar 14;13(1):108. doi: 10.1186/s13287-022-02762-x.
PubMed 35287722
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Outcomes of Autologous Bone Marrow Mononuclear Stem Cell (BMMC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia After Kasai's Operation: An Open Label Uncontrolled Clinical Trial
- ClinicalTrials.gov ID
- NCT03468699
- Acronym / sponsor ID
- VinmecRISCGT78
- Conditions
- Liver Cirrhosis, Biliary
- Keywords
- Autologous bone marrow Transplantation, biliary atresia, Kasai's operation, liver cirrhosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Vinmec Research Institute of Stem Cell and Gene TechnologyUniversity, hospital or other
- Study start
- 2 Jan 2017 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 31 Oct 2021 (actual)
- First posted
- 16 Mar 2018 (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 aim of this study was to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation for Children Suffering from Liver Cirrhosis Due to Biliary Atresia
Detailed description
This study is ferformed to evaluate the safety and effectiveness of autologous bone marrow mononuclear stem cell transplantation in the management of Liver Cirrhosis Due to Biliary Atresia after Kasai's operation of 20 patients at Vinmec Research Institute of Stem Cell and Gene Technology, Hanoi, Vietnam from January 2017 to December 2018
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous BMMC transplantation | Experimental | Combination Product: Autologous BMMC transplantationStem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward |
Who may qualify
- Ages
- 1 year to 15 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Children were 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 after Kasai's operation: hepatomegaly, congestive splenomegaly, elevated liver enzymes, Esophageal Varices (based on Endoscopic Diagnosis), cirrhosis (based on liver biopsy)
Key exclusion criteria (7 listed)
- Under 1 year old patients
- Epilepsy
- Coagulation disorders
- Allergy to anesthetic agents
- Severe health conditions such as cancer, failure of heart, lung, liver or kidney
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 transplantationStem cell transplantation | Combination product | Autologous BMMC transplantation |
Primary outcome
Assess the changes in cholestasis
Time frame: Baseline, 3, 6 months and 12 months after transplantation
Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis
Assess the changes in Liver function
Time frame: Baseline, 3, 6 months and 12 months after transplantation
Using AST (Aspart transaminase) and ALT (Alanin transaminase) (units: U/L), GGT (Gamma GT) (units: UI/L), and Bilirubin (units: mg/dL) to measure the changes in Liver function
Assess the changes in level of cirrhosis
Time frame: Baseline, 3, 6 months and 12 months after transplantation
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 Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).
Assess the changes in liver biopsy
Time frame: Baseline, 3, 6 months and 12 months after transplantation
Liver biopsy, in combination with history and physical examination data, is a powerful clinical tool for diagnosing and treating liver disease to evaluate the changes in liver biopsy
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 4 Jun 2024.
- Background: PubMed 28536370Nicolas C, Wang Y, Luebke-Wheeler J, Nyberg SL. Stem Cell Therapies for Treatment of Liver Disease. Biomedicines. 2016 Jan 6;4(1):2. doi: 10.3390/biomedicines4010002.
- Background: PubMed 17554512Levicar N, Dimarakis I, Flores C, Tracey J, Gordon MY, Habib NA. Stem cells as a treatment for chronic liver disease and diabetes. Handb Exp Pharmacol. 2007;(180):243-62. doi: 10.1007/978-3-540-68976-8_11.
- Background: PubMed 26155042Irfan A, Ahmed I. Could Stem Cell Therapy be the Cure in Liver Cirrhosis? J Clin Exp Hepatol. 2015 Jun;5(2):142-6. doi: 10.1016/j.jceh.2014.03.042. Epub 2014 Mar 27.
- Background: PubMed 20857300Sharma S, Kumar L, Mohanty S, Kumar R, Datta Gupta S, Gupta DK. Bone marrow mononuclear stem cell infusion improves biochemical parameters and scintigraphy in infants with biliary atresia. Pediatr Surg Int. 2011 Jan;27(1):81-9. doi: 10.1007/s00383-010-2712-4.
- Background: PubMed 18555137Khan AA, Parveen N, Mahaboob VS, Rajendraprasad A, Ravindraprakash HR, Venkateswarlu J, Rao P, Pande G, Narusu ML, Khaja MN, Pramila R, Habeeb A, Habibullah CM. Management of hyperbilirubinemia in biliary atresia by hepatic progenitor cell transplantation through hepatic artery: a case report. Transplant Proc. 2008 May;40(4):1153-5. doi: 10.1016/j.transproceed.2008.03.110.
- Derived from this study: PubMed 35287722Nguyen TL, Nguyen HP, Ngo DM, Ha THT, Mai KA, Bui TH, Nguyen PV, Pham LH, Hoang DM, Cao ADT. Autologous bone marrow mononuclear cell infusion for liver cirrhosis after the Kasai operation in children with biliary atresia. Stem Cell Res Ther. 2022 Mar 14;13(1):108. doi: 10.1186/s13287-022-02762-x.
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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 2 study testing Autologous BMMC transplantation in people with Liver Cirrhosis, Biliary, 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 4 inclusion and 7 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 4 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 phase mean?
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?
Assess the changes in cholestasis (time frame: Baseline, 3, 6 months and 12 months after transplantation). The study lists 4 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 NCT03468699, which also lists 6 linked publications.
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 4 Jun 2024; AzTrials last refreshed its copy on 31 Oct 2026.