Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,000
- 试验地点
- 32
- 主要终点
- Change in disease severity over time (disease progression)
研究概览
简要总结
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.
详细描述
This is a multi-center project to establish a prospective database of clinical information and a repository of blood, from children with diagnosis of neonatal liver disease, such as biliary atresia (BA), in order to perform research in this important liver problem. Children (diagnosed with BA or suspicious for BA) will be screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with BA will be followed intensively for the first year, at 18 months of age, and then annually up to 10 years of age, and then biannually, or liver transplantation. Other participants (Non-BA) diagnosed with cholestasis will be exited from the study at the time of diagnosis determination.
Detailed clinical data, laboratory investigations, liver and biliary specimens, and long-term follow-up of outcomes are part of the normal standard of care with respect to the diagnosis and treatment of the subjects with liver problems. This research involves the collection of diagnostic, clinical and outcome data concerning the subject, which is kept without identification (coded) in a national research database of infants with liver disease. Samples of blood will be obtained for later research analysis, whenever possible, at the time of clinically indicated blood draws or when there is IV access for a clinical procedure. All data from this study will be kept in a secure research database at the Scientific Data Coordinating Center (SDCC) and transferred to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) data repository after the study ends.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Infant's age less than or equal to 180 days at initial presentation at the ChiLDReN clinical site.
- •Diagnosis of cholestasis defined by serum direct or conjugated bilirubin greater than or equal to 2 mg/dl and suspected biliary atresia.
- •The subject's parent(s)/guardian(s) willing to provide informed written consent.
排除标准
- •Acute liver failure.
- •Previous hepatobiliary surgery with dissection or excision of biliary tissue.
- •Diagnoses of bacterial or fungal sepsis (except where associated with metabolic liver disease)
- •Diagnoses of hypoxia, shock or ischemic hepatopathy within the past two weeks (If the cholestasis persists beyond two weeks of the initiating event, the infant can be enrolled).
- •Diagnosis of any malignancy.
- •Presence of any primary hemolytic disease (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
- •Diagnosis of any drug or Total parenteral nutrition (TPN)-associated cholestasis (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
- •Diagnosis with Extracorporeal membrane oxygenation (ECMO)-associated cholestasis.
- •Birth weight less than 1500g (except when diagnosed with biliary atresia).
结局指标
主要结局
Change in disease severity over time (disease progression)
时间窗: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
Change in disease severity over time (disease progression)
时间窗: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
次要结局
未报告次要终点