Childhood Liver Disease Research Network Data Coordinating Center

儿童肝病研究网络数据协调中心

基本信息

批准号：
10021642
负责人：
JOHN C MAGEE
金额：
$ 275万
依托单位：
UNIVERSITY OF MICHIGAN AT ANN ARBOR
依托单位国家：
美国
项目类别：
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/10021642
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

项目摘要

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 个临床站点和一个单一的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。