Pentoxifylline Therapy in Biliary Atresia

Baylor College of Medicine

Status and phase: Terminated; Phase 2

Conditions: Biliary Atresia

Treatments: Drug: Pentoxifylline

Study type: Interventional

Funder types: Other

Identifiers

NCT01774487

H-31387

Details and patient eligibility

About

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

Full 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.

Enrollment: 17 patients

Sex: All

Ages: Under 180 days old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

17 participants in 1 patient group

Pentoxifylline

Experimental group

Description:

All newly-diagnosed biliary atresia patients fulfilling the study's inclusion criteria will receive oral pentoxifylline, 20 mg/kg/day divided in three doses for a total of 90 days. The hospital pharmacy will create a 20 mg/ml oral pentoxifylline solution using 400 mg pentoxifylline tablets and established compounding recipes.

Treatment:

Drug: Pentoxifylline