A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy in Infants With Biliary Atresia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 141
- 试验地点
- 14
- 主要终点
- The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy
研究概览
简要总结
The Children Liver Disease Research and Education Network (ChiLDREN) is conducting a clinical trial to evaluate whether long-term treatment with corticosteroids improves the outcome of the Kasai or gall-bladder Kasai in infants with biliary atresia. In this clinical trial, ChiLDREN is testing whether corticosteroid therapy following the Kasai will improve bile drainage and long term outcome in infants with biliary atresia. Subjects in this trial must start treatment within 72 hours of the Kasai procedure and be part of a prospective study of the natural history of biliary atresia also being conducted by ChiLDREN (http://www.clinicaltrials.gov/ct/show/NCT00061828?order=3).
详细描述
This is a multi-center randomized, double-blinded, placebo-controlled trial to prospectively determine the efficacy of corticosteroids on the outcome of infants with biliary atresia. The trial will be conducted by the NIDDK-funded network of 15 clinical centers comprising the Biliary Children Liver Disease Research and Education Network (ChiLDREN), whose goal is to study the etiology, pathogenesis, diagnosis, and treatment of infants with biliary atresia. For the trial, our overall hypothesis is that therapy with corticosteroids following portoenterostomy (including gall bladder Kasai procedure) will improve bile drainage and long-term outcome in infants with biliary atresia. This hypothesis will be tested through the following specific aims and hypotheses:
Aim 1: To determine whether corticosteroid therapy decreases serum bilirubin concentration after portoenterostomy.
Aim 2: To determine whether corticosteroid treatment after portoenterostomy will improve outcome as defined by survival without transplantation at 24 months of age.
Aim 3: To determine whether corticosteroid treatment after portoenterostomy will improve growth of infants with biliary atresia.
Aim 4: To determine whether corticosteroid treatment improves biochemical indicators of each of the fat-soluble vitamins after supplementation with standard doses.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- — 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Portoenterostomy or gall bladder Kasai operation for biliary atresia within the previous 72 hours
- •Post-conception age ≥ 36 weeks
- •Weight at enrolment ≥ 2000 gm
- •Written informed consent to participate in the study obtained prior to or within 72 hours of completion of portoenterostomy. (Note: Families of potential subjects may be approached prior to the portoenterostomy.)
排除标准
- •Known immunodeficiency
- •Diabetes mellitus
- •Presence of significant systemic hypertension for age (persistent systolic blood pressure ≥112 mmHg)
- •A serum indirect (unconjugated) bilirubin ≥ 5 mg/dL for infants under 4 weeks of age or ≥ 7 mg/dL for infants between 4 and 8 weeks of age
- •Known sensitivity to corticosteroids
- •Documented bacteremia or other tissue infection which is felt to be clinically relevant
- •Known congenital infection or disease with herpes simplex virus, toxoplasmosis, or cytomegalovirus inclusion disease of the liver
- •Infants whose mother is known to have human immunodeficiency virus infection
- •Infants whose mother is known to be HBsAg or hepatitis C virus positive
- •Infants with other severe concurrent illnesses such as neurological, cardiovascular, pulmonary, metabolic, endocrine, and renal disorders that would interfere with the conduct and results of the study
- •Any other clinical condition that is a contraindication to the use of corticosteroid (e.g., bowel perforation)
- •Infants who have received the live attenuated rotavirus vaccine (e.g., Rotateq) within 5 days prior to proposed administration of study drug
研究组 & 干预措施
Corticosteroids
干预措施: Corticosteroids (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy
时间窗: Measurements will be made at 6 months after portoenterostomy
次要结局
- Serum Total Bilirubin Concentration(Measurements will be made at 3 months after portoenterostomy)
- Survival With Native Liver at 24 Months of Age(Measurements will be made at 24 months of age)
- Total Bilirubin Concentration at 24 Months of Age(At 24 Months of Age)
- Weight Z-Score(HPE until 24 months of age)
- Height Z-Score(HPE to age 24 Months)
- Presence of Ascites at 24 Months(24 Months)
- Total Bilirubin Concentration at 12 Months(12 Months post HPE)
- Presence of Ascites at 12 Months(12 Months)