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

Liver Cirrhosis, Biliary Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03468699
• Other study ID #: VinmecRISCGT78
• Status: Completed
• Phase: Phase 2
• Start date: January 2, 2017
• Est. completion date: October 31, 2021

Study information

• Verified date: June 2024
• Source: Vinmec Research Institute of Stem Cell and Gene Technology
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: October 31, 2021
• Est. primary completion date: September 30, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 1 Year to 15 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Combination Product:
• Autologous BMMC transplantation
Stem cell transplantation

Locations

Country	Name	City	State
Vietnam	Vinmec Research Institute of Stem Cell and Gene Technology	Hanoi
Vietnam	Vinmec Research Institute of Stem Cell and Gene Technology	Hanoi

Sponsors (1)

Lead Sponsor	Collaborator
Vinmec Research Institute of Stem Cell and Gene Technology

Country where clinical trial is conducted

Vietnam, 

References & Publications (5)

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. — View 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. — View 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. — View 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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Assess the changes in cholestasis	Using Bilirubin (total, direct and indirect) (units: mg/dL) to measure the changes in cholestasis	Baseline, 3, 6 months and 12 months after transplantation
Primary	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	Baseline, 3, 6 months and 12 months after transplantation
Primary	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 If PELD> 15: bad results; Albumin (Unit: g / dL), bilirubin (units: mg / dL) and INR (international normalized ratio).	Baseline, 3, 6 months and 12 months after transplantation
Primary	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	Baseline, 3, 6 months and 12 months after transplantation