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

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ClinicalTrials.gov Identifier: NCT03468699
Recruitment Status : Recruiting
First Posted : March 16, 2018
Last Update Posted : November 26, 2018
Sponsor:
Information provided by (Responsible Party):
Vinmec Research Institute of Stem Cell and Gene Technology

Tracking Information
First Submitted Date  ICMJE January 26, 2018
First Posted Date  ICMJE March 16, 2018
Last Update Posted Date November 26, 2018
Actual Study Start Date  ICMJE January 2, 2017
Estimated Primary Completion Date December 31, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 21, 2018)
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: March 15, 2018)
  • 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
  • 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
  • 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
Change History Complete list of historical versions of study NCT03468699 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Autologous Bone Marrow Mononuclear Stem Cell for Children Suffering From Liver Cirrhosis Due to Biliary Atresia
Official Title  ICMJE 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
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
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Liver Cirrhosis, Biliary
Intervention  ICMJE Combination Product: Autologous BMMC transplantation
Stem cell transplantation
Study Arms  ICMJE 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
Intervention: Combination Product: Autologous BMMC transplantation
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 15, 2018)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2018
Estimated Primary Completion Date December 31, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Year to 15 Years   (Child)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Liem Nguyen, PhD, MD 986565015 v.liemnt@vinmec.com
Listed Location Countries  ICMJE Vietnam
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03468699
Other Study ID Numbers  ICMJE VinmecRISCGT78
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vinmec Research Institute of Stem Cell and Gene Technology
Study Sponsor  ICMJE Vinmec Research Institute of Stem Cell and Gene Technology
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Liem Nguyen Vinmec Research Institute of Stem Cell and Gene Technology
PRS Account Vinmec Research Institute of Stem Cell and Gene Technology
Verification Date November 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP