Clinical Trial: Pentoxifylline Therapy in Biliary Atresia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase II Trial of Pentoxifylline in Newly-Diagnosed Biliary Atresia

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

Detailed 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.


Sponsor: Baylor College of Medicine

Current Primary Outcome: Change in serum conjugated bilirubin [ Time Frame: Baseline and after 90 days of therapy ]

Original Primary Outcome: Change in serum conjugated bilirubin [ Time Frame: Start of therapy and after 90 days of therapy ]

Current Secondary Outcome:

  • Change in Weight [ Time Frame: Baseline and after 90 days of therapy ]
  • Change in serum markers [ Time Frame: Baseline and up to two years after therapy finishes ]
    The investigators will track the change in serum liver markers and platelets over the course of two years in patients receiving 90 days of PTX therapy.
  • Change in liver imaging [ Time Frame: Baseline and up to two years after therapy finishes ]
    The investigators will track liver ultrasound changes, including liver and spleen size.
  • Time to liver transplant [ Time Frame: Baseline and up to two years after therapy finishes ]


Original Secondary Outcome:

  • Change in Weight [ Time Frame: Start of therapy and after 90 days of therapy ]
  • Change in serum markers [ Time Frame: Start of therapy and up to 2 years after therapy finishes ]
    We will track the change in serum liver markers and platelets over the course of two years in patients receiving 90 days of PTX therapy.
  • Change in liver imaging [ Time Frame: Start of therapy and up to 2 years after therapy finishes ]
    We will track liver ultrasound changes, including liver and spleen size.
  • Time to liver transplant [ Time Frame: Start of therapy and up to 2 years after therapy finishes ]


Information By: Baylor College of Medicine

Dates:
Date Received: January 22, 2013
Date Started: January 2013
Date Completion: January 2019
Last Updated: July 18, 2016
Last Verified: July 2016