NCT01774487

Pentoxifylline Therapy in Biliary Atresia

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Baylor College of Medicine · Academic / Other
• Sex: All
• Age: 180 Days
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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.

Conditions

• Biliary Atresia

Interventions

Type	Name	Description
DRUG	Pentoxifylline	20 mg/kg/day divided in 3 doses, given orally for 90 days

Timeline

• Start date: 2013-02-04
• Primary completion: 2018-02-07
• Completion: 2023-02-20
• First posted: 2013-01-24
• Last updated: 2024-01-11
• Results posted: 2023-10-12

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study