Autologous Bone Marrow Mononuclear Stem Cell for Children Suffering From Liver Cirrhosis Due to Biliary Atresia

June 3, 2024 updated by: Vinmec Research Institute of Stem Cell and Gene Technology

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

17

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Vietnam
      • Hanoi, Vietnam, 100000
        • Vinmec Research Institute of Stem Cell and Gene Technology
      • Hanoi, Vietnam, 10000
        • Vinmec Research Institute of Stem Cell and Gene Technology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 11 years (Child)

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • 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)

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Autologous BMMC transplantation
Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward
Combination product: Autologous BMMC transplantation
Stem cell transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Baseline, 3, 6 months and 12 months after transplantation
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
Baseline, 3, 6 months and 12 months after transplantation
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).
Baseline, 3, 6 months and 12 months after transplantation
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
Baseline, 3, 6 months and 12 months after transplantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vinmec Research Institute of Stem Cell and Gene Technology

Investigators

  • Principal Investigator: Liem Nguyen, Vinmec Research Institute of Stem Cell and Gene Technology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 2, 2017

Primary Completion (Actual)

September 30, 2021

Study Completion (Actual)

October 31, 2021

Study Registration Dates

First Submitted

January 26, 2018

First Submitted That Met QC Criteria

March 15, 2018

First Posted (Actual)

March 16, 2018

Study Record Updates

Last Update Posted (Estimated)

June 4, 2024

Last Update Submitted That Met QC Criteria

June 3, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VinmecRISCGT78

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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