Clinical Center for Cholestatic Liver Disease in Children

儿童胆汁淤积性肝病临床中心

• 批准号: 8012205
• 负责人: JORGE A. BEZERRA
• 金额: $ 11万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-02-05 至 2011-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8012205
• 关键词: Address; Ancillary Study; Arts; Bile Acid Biosynthesis Pathway; Bile Acids; Biliary Atresia; Biochemistry; Child; Child Care; Childhood; Chronic; Clinical; Clinical Research; Clinical Trials; Data; Data Coordinating Center; Defect; Development; Education; Enrollment; Etiology; Hepatology; Histopathology; Hospitals; Inherited; Leadership; Liver; Liver diseases; Manuscripts; Molecular; Outcome; Pathogenesis; Patients; Pilot Projects; Principal Investigator; Prospective Studies; Protocols documentation; Research; Research Infrastructure; Research Personnel; Resources; Retrospective Studies; Services; Structure; Study Subject; Syndrome; System; Tissues; Training; Work; Writing; base; improved; infrastructure development; innovation; liver transplantation; medical specialties; member; molecular phenotype; new therapeutic target; novel; operation; programs; working group

DESCRIPTION (provided by applicant): We propose to transition our membership status from the Biliary Atresia Research Consortium (BARC) and the Cholestatic Liver Consortium (CLiC) to the new Childhood Liver Disease Research and Education Network (ChiLDREN). Our proposal represents a logical extension of the long-standing commitment of our Center to improve the care of children with chronic liver disease through innovative patient-based research. We have been a charter member of BARC and CLiC since their creation in 2002 and 2004, respectively. We worked collaboratively with consortium investigators to build the infrastructure to conduct patient-based studies on biliary atresia and cholestatic syndromes. Our key contributions included data submission and analysis of two retrospective studies, leadership in the development of a clinical trial, high enrollment and retention of subjects into three prospective studies and one interventional study, completion of an ancillary study of novel molecular phenotypes of biliary atresia, completion of a pilot project on defects in bile acid synthesis, and participation in working groups and committees related to project reviews, writing of manuscripts, and development of core resources. We look forward to significantly contributing to the operation of ChiLDREN through three aims. In Aim 1, we will combine the expertise and resources of BARC and CLiC to form ChiLDREN. This will be done by transitioning the operation of ongoing study protocols to the working structure developed by the Steering Committee and the Data Coordinating Center, develop new study protocols, and continue to enroll subjects into approved studies. In Aim 2, we will promote specialty training, develop two core services (Bile Acid Biochemistry Core and Histopathology Core), and significantly expand access to study subjects by collaborating with investigators in the Hepatology and Liver Transplant Program at the Hospital for Sick Children, Toronto. And in Aim 3, we will use state-of-the-art molecular and cellular systems to study pathogenesis of liver disease and identify novel therapeutic targets for children with biliary atresia and cholestatic syndromes using data and tissue collected by ChiLDREN protocols. Relevance: We propose to become a member of the Childhood Liver Disease Research and Education Network and to contribute to the development of the infrastructure for clinical research in children with chronic liver disease. This infrastructure will facilitate innovative patient-based studies addressing etiology, pathogenesis, and clinical outcome of children with biliary atresia and inherited cholestatic syndromes.

描述（由申请人提供）： 我们建议将我们的会员身份从胆道闭锁研究联盟（BARC）和胆汁淤积性肝财团（CLIC）转变为新的儿童肝病研究和教育网络（儿童）。我们的建议代表了我们中心长期承诺通过基于创新的患者研究来改善慢性肝病儿童的护理的逻辑扩展。自2002年和2004年创建以来，我们一直是Barc和Clic的宪章成员。我们与财团调查员合作，建立了基础设施，以进行基于患者的胆道闭锁和胆汁淤积综合症研究。我们的关键贡献包括对两项回顾性研究的数据提交和分析，在临床试验发展中的领导，对受试者的高度入学和保留为三项前瞻性研究和一项介入性研究，完成了辅助分子表型的辅助研究的辅助研究，对手机缺陷的完成，对bile Acid构成和参与者的参与者的缺陷，在工作中的缺陷和参与者的参与者，以及在工作中的参与者，以及在工作中的参与者，以及在工作中的参与者，以及在工作中的参与者，以及在工作中的参与者，在工作中的参与者和参与者，在工作中的参与者，在工作中的参与者，在工作中的参与者，在工作中的工作，以及在工作中的工作。核心资源的发展。我们期待通过三个目标为儿童的运作做出重大贡献。在AIM 1中，我们将结合BARC和CLIC的专业知识和资源，形成儿童。这将通过将正在进行的研究方案的运作转换为指导委员会和数据协调中心开发的工作结构，制定新的研究方案，并继续将受试者注册到批准的研究中。在AIM 2中，我们将促进专业培训，开发两种核心服务（胆汁酸生物化学核心和组织病理学核心），并通过与多伦多病态儿童医院的肝病学和肝移植计划合作，通过与研究人员合作来大大扩展对研究对象的访问。在AIM 3中，我们将使用最先进的分子和细胞系统来研究肝病的发病机理，并使用儿童方案收集的数据和组织来确定胆道闭锁和胆汁淤积综合征儿童的新型治疗靶标。 相关性：我们建议成为儿童肝病研究和教育网络的成员，并为患有慢性肝病儿童的临床研究基础设施的发展做出贡献。该基础设施将促进基于创新的患者研究，以解决胆道闭锁和遗传性胆汁淤积综合征的儿童的病因，发病机理和临床结果。