Autologous BMNC Infusion for Liver Cirrhosis in Children With BA (ABMNCBA)

Vinmec Research Institute of Stem Cell and Gene Technology

Status and phase: Completed; Phase 1

Conditions: Biliary Atresia

Treatments: Combination Product: Autologous BMMC transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT05517317

ISC_22_88

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 2 months to 15 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Autologous BMMC infusion

Experimental group

Description:

One administration of autologous bone marrow mononuclear cells via the hepatic artery

Treatment:

Combination Product: Autologous BMMC transplantation