Clinical Trials Logo
  1. Home
  2. Primary Biliary Cirrhosis
  3. NCT04522869

Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Biliary Atresia — UCMSCBA  

Primary Biliary Cirrhosis Clinical Trial

Official title:
Evaluation Safety and Efficacy of Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia: A Matched Control Prospective Study

Clinical Trial Summary

Biliary atresia (BA) is the most frequent cause of chronic cholestasis in neonates, accounting for at least 50% of pediatric liver transplantation. BA incidence is estimated to range from 1:5000 to 1:19000 live births. All patients will die due to complications of liver cirrhosis if the operation is not performed. Recently, mesenchymal stem cell (MSC) transplantation has been found as a promising therapy for liver cirrhosis in adults. Bone marrow-derived stem cell transplantation was also performed successfully for children with BA. Compared to MSC isolation from bone marrow, isolating MSCs from umbilical cord (UC) tissue is a less invasive procedure. Furthermore, UC-derived MSCs (UC-MSCs) have been demonstrated to be safe and effective for liver cirrhosis in adults and different pediatric diseases, including liver cirrhosis due to primary biliary cirrhosis. The investigators will compare the outcomes of 17 Kasai operated BA patients who receive UC-MSC transplantation to 17 BA patients who only undergo Kasai operation. Two transplantations of UC - MSCs will be performed via the hepatic artery: the first transplant will be performed at baseline, and the second one will be performed 6 months later with a dosage of 1 million MSCs per kg of body weight. The frequency and severity of the adverse events or serious adverse events associated with UC-MSC injection at 72 hours post-injection will be used to assess the safety. The efficacy of the therapy will be measured using Pediatric End-Stage Liver Disease (PELD) score, liver function, and liver biopsy. This study would open a novel cell therapy to improve outcomes of patients with BA.

Clinical Trial Description

The study protocol was approved by the Vinmec International Hospital Ethics Committee, and National Ethics Committees. The stem cell products are conducted in accordance with (GMP) requirements and Good Clinical Practice (GCP). All patients and primary caregivers will receive a written consent form, a cover letter and a clear explanation of the safety issues, potential risks and benefits, and the procedure involved. Moreover, patients will be provided the updated results related to disease and the study during conducting the study and will be fully funded by the project. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04522869
Study type Interventional
Source Vinmec Research Institute of Stem Cell and Gene Technology
Contact
Status Completed
Phase Phase 1/Phase 2
Start date August 10, 2019
Completion date October 25, 2021
View Details
See also
  Status Clinical Trial Phase
Active, not recruiting NCT04526665 - Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC) Phase 3
Recruiting NCT02931513 - sCD163 in PBC Patients - Assessment of Treatment Response
Active, not recruiting NCT02924701 - sCD163 in PBC Patients - Assessment of Disease Severity and Prognosis
Completed NCT02659696 - Nalfurafine Hydrochloride for Pruritus in Patients With Primary Biliary Cholangitis
Completed NCT02078882 - Study of Abatacept (Orencia) to Treat Primary Biliary Cirrhosis Phase 4
Completed NCT01603199 - High-protein High-fiber Diet in Patients With Primary Biliary Cirrhosis N/A
Completed NCT01389973 - A Study of Efficacy and Safety of Ustekinumab in Patients With Primary Biliary Cirrhosis (PBC) Who Had an Inadequate Response to Ursodeoxycholic Acid Phase 2
Completed NCT01857284 - Safety and Efficacy of Tauroursodeoxycholic Acid Versus Ursofalk in the Treatment of Adult Primary Biliary Cirrhosis Phase 3
Completed NCT05374200 - Mind-body Wellness Intervention in Primary Biliary Cholangitis (PBC) N/A
Recruiting NCT02937012 - Use of Bezafibrate in Patients With Primary Biliary Cirrhosis to Archive Complete Biochemical Response in Non-responders Phase 3
Completed NCT02376335 - B-Cell Depleting Therapy (Rituximab) as a Treatment for Fatigue in Primary Biliary Cirrhosis Phase 2
Recruiting NCT01662973 - Umbilical Cord Mesenchymal Stem Cells for Patients With Primary Biliary Cirrhosis Phase 1/Phase 2
Active, not recruiting NCT04751188 - A Study to Assess Efficacy and Safety of Bezafibrate in Patients With Primary Biliary Cholangitis Phase 3
Recruiting NCT04514965 - Bezafibrate in Patients With Primary Biliary Cholangitis (PBC)
Recruiting NCT03668145 - Mesenchymal Stem Cell Transplantation for Refractory Primary Biliary Cholangitis N/A
Completed NCT02955602 - Seladelpar (MBX-8025) in Subjects With Primary Biliary Cholangitis (PBC) Phase 2
Completed NCT02557360 - Effectiveness of S-adenosyl-L-methionine in Patients With Primary Biliary Cirrhosis Phase 4
Recruiting NCT01440309 - Efficacy and Safety Study of Allogenic Mesenchymal Stem Cells for Patients With Refractory Primary Biliary Cirrhosis Phase 1
Completed NCT01249092 - Pentoxifylline for Primary Biliary Cirrhosis Phase 2
Completed NCT01510860 - Ursofalk Tablets (500 mg) Versus Ursofalk Capsules (250 mg) in the Treatment of Primary Biliary Cirrhosis Phase 4