Clinical trial NCT03468699: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Liver Cirrhosis, Biliary
Keywords: Autologous bone marrow Transplantation
biliary atresia
Kasai's operation
liver cirrhosis
Lead sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
Sponsor class: OTHER
First posted: 2018-03-16
Enrollment: 17
Sex: ALL
Minimum age (years): 1
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility criteria: 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

Primary outcomes:
measure: Assess the changes in cholestasis
timeFrame: Baseline, 3, 6 months and 12 months after transplantation
measure: Assess the changes in Liver function
timeFrame: Baseline, 3, 6 months and 12 months after transplantation
measure: Assess the changes in level of cirrhosis
timeFrame: Baseline, 3, 6 months and 12 months after transplantation
measure: Assess the changes in liver biopsy
timeFrame: Baseline, 3, 6 months and 12 months after transplantation
Secondary outcomes:
Other outcomes:
Locations:
city: Hanoi
country: Vietnam
facility: Vinmec Research Institute of Stem Cell and Gene Technology
city: Hanoi
country: Vietnam
facility: Vinmec Research Institute of Stem Cell and Gene Technology
Study officials:
affiliation: Vinmec Research Institute of Stem Cell and Gene Technology
name: Liem Nguyen
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.
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.
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.
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.
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.