Pentoxifylline Therapy in Biliary Atresia
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Trial of Pentoxifylline in Newly-Diagnosed Biliary Atresia|
- Change in serum conjugated bilirubin [ Time Frame: Baseline and after 90 days of therapy ] [ Designated as safety issue: No ]
- Change in Weight [ Time Frame: Baseline and after 90 days of therapy ] [ Designated as safety issue: No ]
- Change in serum markers [ Time Frame: Baseline and up to two years after therapy finishes ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]
|Study Start Date:||January 2013|
|Estimated Study Completion Date:||January 2019|
|Estimated Primary Completion Date:||January 2017 (Final data collection date for primary outcome measure)|
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.
20 mg/kg/day divided in 3 doses, given orally for 90 days
Other Name: Trental
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01774487
|Contact: Sanjiv Harpavat, MD PhD||832-824-2099 ext email@example.com|
|Contact: Ross Shepherd, MD||832-824-2099 ext 1223||Ross.Shepherd@bcm.edu|
|United States, Texas|
|Texas Children's Hospital and Baylor College of Medicine||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Sanjiv Harpavat, MD PhD 832-824-2099 ext 2144 firstname.lastname@example.org|
|Contact: Ross Shepherd, MD 832-824-2099 ext 1223 Ross.Shepherd@bcm.edu|
|Principal Investigator: Sanjiv Harpavat, MD PhD|
|Principal Investigator: Ross Shepherd, MD|
|Sub-Investigator: Mary Brandt, MD|
|Sub-Investigator: Shelly Kim, PharmD|
|Sub-Investigator: Charles Minard, PhD|
|Sub-Investigator: Paula Hertel, MD|
|Sub-Investigator: Milton Finegold, MD|
|Principal Investigator:||Sanjiv Harpavat, MD PhD||Baylor College of Medicine|
|Principal Investigator:||Ross Shepherd, MD||Baylor College of Medicine|