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

Primary Purpose

Liver Cirrhosis, Biliary

Status

Unknown status

Phase

Phase 2

Locations

Vietnam

Study Type

Interventional

Intervention

Autologous BMMC transplantation

About this trial

This is an interventional treatment trial for Liver Cirrhosis, Biliary focused on measuring Autologous bone marrow Transplantation, biliary atresia, Kasai's operation, liver cirrhosis

Eligibility Criteria

1 Year - 15 Years (Child)All SexesAccepts Healthy Volunteers

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

Sites / Locations

Vinmec Research Institute of Stem Cell and Gene TechnologyRecruiting
Vinmec Research Institute of Stem Cell and Gene TechnologyRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous BMMC transplantation

Arm Description

Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward

Outcomes

Primary Outcome Measures

Assess the changes in cholestasis

Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis

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

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).

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

Secondary Outcome Measures

Full Information

NCT ID

NCT03468699

First Posted

January 26, 2018

Last Updated

August 17, 2020

Sponsor

Vinmec Research Institute of Stem Cell and Gene Technology

1. Study Identification

Unique Protocol Identification Number

NCT03468699

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2019

Overall Recruitment Status

Unknown status

Study Start Date

January 2, 2017 (Actual)

Primary Completion Date

September 30, 2020 (Anticipated)

Study Completion Date

October 31, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vinmec Research Institute of Stem Cell and Gene Technology

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Liver Cirrhosis, Biliary

Keywords

Autologous bone marrow Transplantation, biliary atresia, Kasai's operation, liver cirrhosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Autologous BMMC transplantation

Arm Type

Experimental

Arm Description

Stem cell transplantations include 2 administrations of autologous bone marrow mononuclear cells via the hepatic artery at baseline and 6 months afterward

Intervention Type

Combination Product

Intervention Name(s)

Autologous BMMC transplantation

Intervention Description

Stem cell transplantation

Primary Outcome Measure Information:

Title

Assess the changes in cholestasis

Description

Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis

Time Frame

Baseline, 3, 6 months and 12 months after transplantation

Title

Assess the changes in Liver function

Description

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

Time Frame

Baseline, 3, 6 months and 12 months after transplantation

Title

Assess the changes in level of cirrhosis

Description

Time Frame

Baseline, 3, 6 months and 12 months after transplantation

Title

Assess the changes in liver biopsy

Description

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

Time Frame

Baseline, 3, 6 months and 12 months after transplantation

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Year

Maximum Age & Unit of Time

15 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Liem Nguyen, PhD, MD

Phone

986565015

v.liemnt@vinmec.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Liem Nguyen

Organizational Affiliation

Vinmec Research Institute of Stem Cell and Gene Technology

Official's Role

Principal Investigator

Facility Information:

Facility Name

Vinmec Research Institute of Stem Cell and Gene Technology

City

Hanoi

ZIP/Postal Code

100000

Country

Vietnam

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Liem T Nguyen, MD., PhD

Phone

(+844)39743556

Ext

1420

v.liemnt@vinmec.com

Facility Name

Vinmec Research Institute of Stem Cell and Gene Technology

City

Hanoi

ZIP/Postal Code

10000

Country

Vietnam

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Phuong Nguyen, MPH

Phone

(+84)914740683

hoangphuonghsph@gmail.com

First Name & Middle Initial & Last Name & Degree

Liem Nguyen, Prof

12. IPD Sharing Statement

Citations:

PubMed Identifier

28536370

Citation

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.

Results Reference

background

PubMed Identifier

17554512

Citation

Levicar 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.

Results Reference

background

PubMed Identifier

26155042

Citation

Irfan 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.

Results Reference

background

PubMed Identifier

20857300

Citation

Sharma 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.

Results Reference

background

PubMed Identifier

18555137

Citation

Khan 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.

Results Reference

background

PubMed Identifier

35287722

Citation

Nguyen 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.

Results Reference

derived

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

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