ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03468699
Recruitment Status : Recruiting
First Posted : March 16, 2018
Last Update Posted : March 16, 2018
Sponsor:
Information provided by (Responsible Party):
Vinmec Research Institute of Stem Cell and Gene Technology

Brief 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

Condition or disease Intervention/treatment Phase
Liver Cirrhosis, Biliary Combination Product: Autologous BMMC transplantation Phase 2

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  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : January 2, 2017
Estimated Primary Completion Date : December 31, 2018
Estimated Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cirrhosis

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




Primary Outcome Measures :
  1. Assess the changes in cholestasis [ Time Frame: Baseline, 3, 6 months and 12 months after transplantation ]
    Using Bilirubin (total, direct and indirect) to measure the changes in cholestasis

  2. 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

  3. 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

  4. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
  • 1 year old or older
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03468699


Contacts
Contact: Liem Nguyen, PhD, MD 986565015 v.liemnt@vinmec.com

Locations
Vietnam
Vinmec Research Institute of Stem Cell and Gene Technology Recruiting
Hanoi, Vietnam, 100000
Contact: Liem T Nguyen, MD., PhD    (+844)39743556 ext 1420    v.liemnt@vinmec.com   
Vinmec Research Institute of Stem Cell and Gene Technology Recruiting
Hanoi, Vietnam, 10000
Contact: Phuong Nguyen, MPH    (+84)914740683    hoangphuonghsph@gmail.com   
Principal Investigator: Liem Nguyen, Prof         
Sponsors and Collaborators
Vinmec Research Institute of Stem Cell and Gene Technology
Investigators
Principal Investigator: Liem Nguyen Vinmec Research Institute of Stem Cell and Gene Technology

Publications:
Responsible Party: Vinmec Research Institute of Stem Cell and Gene Technology
ClinicalTrials.gov Identifier: NCT03468699     History of Changes
Other Study ID Numbers: VinmecRISCGT78
First Posted: March 16, 2018    Key Record Dates
Last Update Posted: March 16, 2018
Last Verified: March 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Vinmec Research Institute of Stem Cell and Gene Technology:
Autologous bone marrow Transplantation
biliary atresia
Kasai's operation
liver cirrhosis

Additional relevant MeSH terms:
Liver Cirrhosis
Liver Cirrhosis, Biliary
Fibrosis
Biliary Atresia
Pathologic Processes
Liver Diseases
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Abnormalities
Congenital Abnormalities
Cholestasis, Intrahepatic
Cholestasis