Houston, Texas 77030

  • Biliary Atresia


The purpose of this study is to determine whether pentoxifylline reduces liver damage in infants with biliary atresia.

Study summary:

Biliary atresia (BA) is a devastating liver disease of infancy of unknown etiology, characterized by bile duct obstruction, live fibrosis, and cirrhosis. BA has no known medical treatments. The only proven treatment is a surgical portoenterostomy (the Kasai procedure, or KP) which can achieve bile drainage and improve outcomes in some cases. The KPs success is variable depending on several factors including age of the infant, experience of the surgeon, and extent of liver fibrosis at the time of KP. In this study, the investigators conduct a phase II trial of a potential new medical therapy for BA: pentoxifylline (PTX). PTX is a methylxanthine derivative closely related to caffeine that has been used safely in infants with other diseases such as sepsis. In adults, PTX has been shown to have a number of properties beneficial to the liver, including preventing liver fibrosis, improving liver regeneration, and reducing cirrhosis-related complications. The trial's objective is to determine whether PTX has sufficient biological activity against BA to warrant further study. PTX will be administered orally for 90 days as an adjunct to standard therapy (i.e. KP if appropriate). The primary outcome will measure the change in serum conjugated bilirubin levels after 90 days. Secondary outcomes include changes in body weight, serum markers, liver imaging, and time to liver transplant in infants with BA.


Inclusion Criteria: - 0-180 days old - Diagnosed with biliary atresia through liver biopsy and/or intra-operative cholangiogram - No previous Kasai portoenterostomy performed at another institution - Able to take medications orally - Legal guardian signs consent after understanding risks and investigational nature of study Exclusion Criteria: - Infants greater than 180 days old - Infants receiving a Kasai portoenterostomy at another institution - Infants unable to take medications orally



Primary Contact:

Principal Investigator
Sanjiv Harpavat, MD PhD
Baylor College of Medicine

Sanjiv Harpavat, MD PhD
Phone: 832-824-2099 ext. 2144
Email: harpavat@bcm.edu

Backup Contact:

Email: Ross.Shepherd@bcm.edu
Ross Shepherd, MD
Phone: 832-824-2099 ext. 1223

Location Contact:

Houston, Texas 77030
United States

Sanjiv Harpavat, MD PhD
Phone: 832-824-2099
Email: harpavat@bcm.edu

Site Status: Recruiting

Data Source: ClinicalTrials.gov

Date Processed: June 17, 2021

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