Clinical trial NCT01774487: Pentoxifylline Therapy in Biliary Atresia
Official title: A Phase II Trial of Pentoxifylline in Newly-Diagnosed Biliary Atresia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Biliary Atresia
Keywords: Biliary Atresia
Pentoxifylline
Trental
Serum bilirubin
Conjugated bilirubin
Liver transplantation
Fibrosis
Lead sponsor: Baylor College of Medicine
Sponsor class: OTHER
First posted: 2013-01-24
Enrollment: 17
Sex: ALL
Maximum age (years): 180
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: 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 outcomes:
measure: Number of Participants With Normal Serum Conjugated Bilirubin Levels 12 Weeks After Starting PTX (Pentoxifylline) Therapy
timeFrame: 12 weeks after starting therapy
Secondary outcomes:
measure: Number of Participants Achieving Zero or Positive Weight Z-scores 12 Weeks After Starting PTX Therapy
timeFrame: 12 weeks after starting therapy
measure: Alanine Amino Transferase (ALT) Levels at 2 Years of Life
timeFrame: 2 years of age
measure: Spleen Size at 2 Years of Age
timeFrame: 2 years of age
measure: Time to Liver Transplant
timeFrame: Baseline and up to two years after therapy finishes
measure: Platelet Levels at 2 Years of Life
timeFrame: 2 years of age
Other outcomes:
Locations:
city: Houston
country: United States
facility: Texas Children's Hospital and Baylor College of Medicine
state: Texas
Study officials:
affiliation: Baylor College of Medicine
name: Sanjiv Harpavat, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
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