Childhood Liver Disease Research Network Data Coordinating Center

儿童肝病研究网络数据协调中心

• 批准号: 10251471
• 负责人: JOHN C MAGEE
• 金额: $ 90.94万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-18 至 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10251471
• 关键词: Alagille Syndrome; Ancillary Study; Bar Codes; Bile Acid Biosynthesis Pathway; Biliary Atresia; Case Report Form; Charge; Child; Childhood; Chronic; Clinical Research; Clinical Trials Data Monitoring Committees; Clinical Trials Design; Collaborations; Communication; Data; Data Analyses; Data Collection; Data Coordinating Center; Defect; Development; Disease; Disease model; Family; Funding; Genomics; Goals; Human Resources; Infrastructure; Institutional Review Boards; Intervention Studies; Knowledge; Lead; Leadership; Learning; Liver Fibrosis; Liver diseases; Logistics; Manuscripts; Mitochondria; Monitor; Morbidity - disease rate; National Institute of Diabetes and Digestive and Kidney Diseases; Natural History; Observational Study; Pathogenesis; Patient Care; Patients; Phase; Policies; Process; Progressive intrahepatic cholestasis; Protocols documentation; Publications; Reporting; Research; Research Design; Research Personnel; Sampling; Scientist; Secure; Site; Societies; Statistical Data Interpretation; Syndrome; System; Testing; Time; Translational Research; United States; Work; Writing; alpha 1-Antitrypsin Deficiency; base; biomarker development; cholestatic liver disease; chronic liver disease; clinical research site; data management; design; disease diagnosis; dissemination research; insight; liver cystic fibrosis; liver transplantation; meetings; neonatal hepatitis; primary endpoint; primary sclerosing cholangitis; protocol development; quality assurance; research study; response; secondary endpoint; symposium; tool; validation studies; web site; working group; young adult

Project Summary Abstract Chronic pediatric liver disease is a devastating group of conditions that have a profound effect on children, their families, and our society. Biliary atresia and the other cholestatic liver diseases studied by this network account for over half of liver transplants performed in children in the United States. A better understanding of the diseases studied by this network will help provide better care of patients with chronic liver disease. The gaps in our current knowledge regarding these diseases provide compelling scientific justification for the continuation of the Childhood Liver Disease Research Network (ChiLDReN). As stated in the RFA, the primary goal of this proposal is to continue clinical and translational research on rare pediatric liver diseases that include: biliary atresia; Alagille syndrome; alpha-1-antitrypsin deficiency; progressive familial intrahepatic cholestasis syndromes; bile acid synthesis defects; mitochondrial hepatopathies; idiopathic neonatal hepatitis; cystic fibrosis liver disease; and primary sclerosing cholangitis. It is anticipated that the network will consist of up to 13 clinical sites and a single scientific and data coordinating center (SDCC). The SDCC for the ChiLDReN study is charged with providing coordination, communications and logistical support, clinical study design, centralized data management, biosample management, quality assurance, and analytical support to the research sites and the NIDDK Project Scientist for all ChiLDReN studies. The proposing team is submitting this response to RFA-DK-18-502 to continue as the SDCC for the network.

项目概要 摘要 慢性小儿肝病是一组毁灭性的疾病，对儿童产生深远的影响， 他们的家庭，以及我们的社会。该网络研究的胆道闭锁和其他胆汁淤积性肝病 占美国儿童肝移植的一半以上。更好地理解 该网络研究的疾病将有助于为慢性肝病患者提供更好的护理。这 我们目前对这些疾病的了解存在差距，这为以下观点提供了令人信服的科学依据： 儿童肝病研究网络 (ChiLDReN) 的延续。 正如 RFA 中所述，该提案的主要目标是继续对罕见疾病进行临床和转化研究。 小儿肝脏疾病，包括： 胆道闭锁；阿拉吉尔综合症; α-1-抗胰蛋白酶缺乏症； 进行性家族性肝内胆汁淤积综合征；胆汁酸合成缺陷；线粒体 肝病;特发性新生儿肝炎；囊性纤维化肝病；和原发性硬化性胆管炎。它 预计该网络将由多达 13 个临床站点和一个科学和数据协调中心组成 中心（SDCC）。 ChiLDReN 研究的 SDCC 负责提供协调、沟通 和后勤支持、临床研究设计、集中数据管理、生物样本管理、质量 为所有儿童的研究地点和 NIDDK 项目科学家提供保证和分析支持 研究。提案团队正在向 RFA-DK-18-502 提交此回复，以继续作为 SDCC 网络。