Clinical trial NCT05517317: Autologous BMNC Infusion for Liver Cirrhosis in Children With BA
Official title: Autologous Bone Marrow Mononuclear Cell Infusion for Liver Cirrhosis in Children With Biliary Atresia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Biliary Atresia
Lead sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
Sponsor class: OTHER
First posted: 2022-08-26
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Autologous BMMC infusion
Primary purpose: TREATMENT

Brief summary: To evaluate the safety and early outcomes of autologous bone marrow mononuclear cell (BMMNC) infusion for liver cirrhosis due to biliary atresia (BA) after Kasai operation. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery.

Detailed description: Biliary atresia (BA) is a progressive fibro-obliterative cholangiopathy and a fatal disease. Without surgery, children with BA rarely survive beyond three years of age. The reported prevalence of BA ranges from 1 in 9640 to 1 in 19,500 live births. In the past, most children with a "non-correctable" type of BA died without adequate treatment. Recently, stem cell administration has been applied in adults with liver cirrhosis and has shown promising outcomes. An open-label clinical trial was performed from January 2015 to December 2021. 12 children with liver cirrhosis due to BA at the time of Kasai or after Kasai were included. Bone marrow was harvested through anterior iliac crest puncture under general anesthesia. Mononuclear cells (MNCs) were isolated by Ficoll gradient centrifugation and then infused into the hepatic artery. Serum bilirubin, albumin, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, and prothrombin time were monitored at baseline, three months, six months and 12 months after the transplantation. Esophagoscopies and liver biopsies were performed in patients whose parents provided consent. This study aimed to evaluate both safety and hepatic function after BMMNC administration in these children.

Eligibility criteria: Inclusion Criteria:

* Children were diagnosed with Liver Cirrhosis Due to biliary atresia after Kasai's operation
* Two months old or older
* Patients with a 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:

* 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: Adverse events and serious adverse events
timeFrame: up to the 12-month period following treatment
Secondary outcomes:
measure: The changes in cholestasis
timeFrame: up to the 12-month period following treatment
measure: The changes in Liver function using Aspart transaminase
timeFrame: up to the 12-month period following treatment
measure: The changes in Liver function using Alanine transaminase
timeFrame: up to the 12-month period following treatment
measure: The changes in Liver function using Gamma GT
timeFrame: up to the 12-month period following treatment
measure: The changes in level of cirrhosis
timeFrame: up to the 12-month period following treatment
measure: The changes in liver biopsy
timeFrame: up to the 12-month period following treatment
Other outcomes:
Locations:
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 Thanh Nguyen, PhD
role: PRINCIPAL_INVESTIGATOR
References: