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

Primary Biliary Cirrhosis Clinical Trial

— UCMSCBA  

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 Details — Status: Recruiting

Administrative data

• NCT number: NCT04522869
• Other study ID #: ISC.19.10
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 10, 2019
• Est. completion date: October 25, 2021

Study information

• Verified date: August 2020
• Source: Vinmec Research Institute of Stem Cell and Gene Technology
• Contact: Phuong Nguyen
• Phone: (+84) 243 975
• Email: v.phuongnh9[@]vinmec.com
• Is FDA regulated: No
• Study type: Interventional

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 34
• Est. completion date: October 25, 2021
• Est. primary completion date: September 25, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Months to 2 Years

Eligibility

Inclusion Criteria:

• Children 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 on liver biopsy during Kasai's operation.

• Parents or primary caregivers signed the informed consent form.

Exclusion Criteria:

• Under 6kg or over 2 years old

• Coagulation disorders

• Allergy to anesthetic agents

• Active infections

Related Conditions & MeSH terms

• Biliary Atresia
• Fibrosis
• Liver Cirrhosis
• Liver Cirrhosis, Biliary
• Primary Biliary Cirrhosis

Intervention

Biological:
• Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation
Umbilical Cord Derived Mesenchymal Stem Cell (UC -MSC) Transplantation for Children Suffering From Liver Cirrhosis Due to Biliary Atresia

Locations

Country	Name	City	State
Vietnam	Vinmec International Hospital Times City	Hà N?i	Hanoi
Vietnam	Vinmec Research Institute of Stem cell and Gene Technology	Hanoi

Sponsors (2)

Lead Sponsor	Collaborator
Vinmec Research Institute of Stem Cell and Gene Technology	Children's Hospital Number 2, Ho Chi Minh City, Vietnam

Country where clinical trial is conducted

Vietnam, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The change of PELD scores during study	The PELD score is calculated using the following formula: PELD Score = 0.480 * ln (Bilirubin in mg/dL) + 1.857 * ln (INR) - 0.687 * ln (Albumin in g/dL) + 0.436 if the patient is <1 year old + 0.667 if there growth failure	baseline, 3 months, 6 months, 9 months, 12 months
Primary	The change of albumin (Liver function)	Levels of albumin (g/dL)	up to the 12-month period following treatment
Primary	The change of total bilirubin (Liver function)	Total bilirubin level (mg/dL)	up to the 12-month period following treatment
Primary	The change of prothrombin time	Prothrombin time (second)	up to the 12-month period following treatment
Primary	The change of liver biopsy	change of liver biopsy	up to the 12-month period following treatment
Secondary	The number of Adverse Events (AE) and Serious Adverse Events (SAE)	Adverse Events (AE) and Serious Adverse Events (SAE) after MSC transplantation	baseline, 3 months, 6 months, 9 months, 12 months (time frame: up to the 12-month period following treatment)