Primary Biliary Cirrhosis Clinical Trial
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
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.
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. ;
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