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

Phase 2

Completed

• Conditions: Liver Cirrhosis, Biliary
• Interventions: Combination Product: Autologous BMMC transplantation

• Registration Number: NCT03468699
• Lead Sponsor: 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

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 Timeline

• Study Start: 2017-01-02
• Study First Submitted: 2018-01-26
• Study First Submitted QC: 2018-03-15
• Study First Posted: 2018-03-16
• Primary Completion: 2021-09-30
• Study Completion: 2021-10-31
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-03
• Last Update Posted: 2024-06-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous BMMC transplantation	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

Primary Outcome Measures

Name	Time	Method
Assess the changes in liver biopsy	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
Assess the changes in cholestasis	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	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	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).

Trial Locations

Locations (1)

Vinmec Research Institute of Stem Cell and Gene Technology; 🇻🇳 Hanoi, Vietnam