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: 9129366
• 负责人: JOHN C MAGEE
• 金额: $ 82.86万
• 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 2019-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9129366
• 关键词: Accounting; Alagille Syndrome; Area; Bile Acid Biosynthesis Pathway; Biliary Atresia; Biometry; Charge; Child; Childhood; Chronic; Clinical Research; Clinical Trials Data Monitoring Committees; Collection; Communication; Core Facility; Cystic Fibrosis; Data; Data Base Management; Data Coordinating Center; Defect; Deposition; Disease; Family; Goals; Health; Investigational New Drug Application; Knowledge; Laboratories; Learning; Life; Liver diseases; Mitochondria; Monitor; National Institute of Diabetes and Digestive and Kidney Diseases; Patient Care; Patients; Progressive intrahepatic cholestasis; Publications; Report (document); Research; Research Design; Research Infrastructure; Research Personnel; Scientist; Services; Site; Societies; Syndrome; Testing; Translational Research; Transplant-Related Disorder; United States; United States Food and Drug Administration; acronyms; alpha 1-Antitrypsin Deficiency; chronic liver disease; clinical research site; data management; database design; disease diagnosis; dissemination research; experience; improved; liver transplantation; neonatal hepatitis; quality assurance; repository; response; young adult

DESCRIPTION (provided by applicant): 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 ChiLDReN. As stated in the RFA, the primary goal of this proposal is to continue clinical and translational research on rare pediatric liver disease that include: biliary atresia; Alagille syndrome, alpha-1-antitrypsin deficiency, progressive familial intrahepatic cholestasis syndromes, bile acid synthesis defects, mitochondrial hepatopathies, idiopathic neonatal hepatitis, and cystic fibrosis liver disease. It is anticipated that the network will consist of up to 15 clinical sites and a single data coordinating center (DCC). The DCC 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-13-011 to continue as the DCC for the network.

描述（由申请人提供）：慢性儿童肝病是一组对儿童，他们的家庭和我们的社会产生深远影响的破坏性疾病。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脏移植手术的一半以上。更好地了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。我们目前对这些疾病的知识存在差距，这为《儿童》的继续提供了令人信服的科学依据。如RFA所述，该提案的主要目标是继续对罕见儿科肝病的临床和转化研究，包括：胆道闭锁；Alagille综合征、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、特发性新生儿肝炎和囊性纤维化肝病。预计该网络将由多达15个临床站点和一个数据协调中心（DCC）组成。儿童研究的DCC负责为所有儿童研究的研究地点和NIDDK项目科学家提供协调、通信和后勤支持、临床研究设计、集中数据管理、生物样本管理、质量保证和分析支持。提案小组正在向RFA-DK-13-011提交此响应，以继续作为网络的DCC。