A Phase II Trial of Pentoxifylline in Newly-Diagnosed Biliary Atresia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Number of Participants With Normal Serum Conjugated Bilirubin Levels 12 Weeks After Starting PTX (Pentoxifylline) Therapy
研究概览
简要总结
The purpose of this study is to determine whether pentoxifylline reduces liver damage in infants with biliary atresia.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 180 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •Infants greater than 180 days old
- •Infants receiving a Kasai portoenterostomy at another institution
- •Infants unable to take medications orally
研究组 & 干预措施
Pentoxifylline
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.
干预措施: Pentoxifylline (Drug)
结局指标
主要结局
Number of Participants With Normal Serum Conjugated Bilirubin Levels 12 Weeks After Starting PTX (Pentoxifylline) Therapy
时间窗: 12 weeks after starting therapy
The investigators will track the serum conjugated bilirubin (CB) levels over the course of therapy in patients receiving 90 days of PTX (this laboratory test is drawn as part of routine care). Normal CB is 0.0-0.3 mg/dL, with a higher number of patients meeting this indicating a better outcome.
次要结局
- Number of Participants Achieving Zero or Positive Weight Z-scores 12 Weeks After Starting PTX Therapy(12 weeks after starting therapy)
- Alanine Amino Transferase (ALT) Levels at 2 Years of Life(2 years of age)
- Platelet Levels at 2 Years of Life(2 years of age)
- Spleen Size at 2 Years of Age(2 years of age)
- Time to Liver Transplant(Baseline and up to two years after therapy finishes)
研究者
Sanjiv Harpavat
Assistant Professor, Department of Pediatrics, Division of Gastroenterology, Hepatology, and Nutrition
Baylor College of Medicine