NASH Clinical Research Network

描述(由申请人提供)：本申请是对非酒精性脂肪性肝炎临床研究网络(NASH CRN)-数据协调中心的延续。非酒精性脂肪性肝病(NAFLD)在北美每三个成年人和五个儿童中就有一个受到影响，在美国是一个日益严重的公共卫生问题。非酒精性脂肪肝，尤其是非酒精性脂肪性肝炎(NASH)，可能导致终末期肝病和原发性肝癌，以及与肝脏、心血管和癌症相关的死亡，导致医疗负担和成本的大幅增加。NASH CRN是影响NASH日益增长的公共卫生意义的理想和独特的定位，只能通过更大的研究联盟来解决。NASH CRN的主要目标是在成人和儿童中进行NASH和NAFLD的临床研究。一个密切联系和高度优先的次要目标是在NASH和NAFLD中进行转译研究，重点放在发病机制上，这将为了解NASH和NAFLD的自然历史和开发更好的诊断、治疗和临床管理手段提供基础。在NASH CRN的下一阶段，将完成在前一个供资周期启动的成人和儿童治疗试验，以及 将启动新的治疗试验，包括2a阶段机制证明和2b阶段临床试验，以开发安全、有效、简单和廉价的循证治疗方案。成人和儿童NAFLD的纵向队列将扩大，这将前瞻性地定义疾病的自然病史、心血管和代谢风险 这将有助于生物标记物的发现和验证，以及非侵入性技术的开发和验证，以评估和识别NASH/NAFLD患者、谁将做出反应以及疾病进展的速度。杜克大学将为NASH CRN提供NAFLD方面的专业基础科学和临床翻译专业知识，能够轻易地招募成人和儿童参加预期的临床试验，并进行辅助研究，以填补现有的知识空白。杜克大学和我们的儿科网站Ann&Robert H.Lurie儿童医院/JHU渴望继续我们与NIDDK项目工作人员、数据协调中心、私营部门合作伙伴和其他临床中心的合作，以完成NASH CRN的重要研究目标。NASH CRN准备继续其在该领域的主要影响，并直接推动国家卫生研究院改善公众健康的使命。

DESCRIPTION (provided by applicant): This application is for the Continuation of the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) - Data Coordinating Center. Nonalcoholic fatty liver disease (NAFLD) affects one out of every three adults and five children in North America and is a growing public health issue in the United States. NAFLD, and especially nonalcoholic steatohepatitis (NASH), may lead to end-stage liver disease and primary liver cancer, as well as liver-, cardiovascular-, and cancer-related mortality, resulting in major increases in health burdens and costs. The NASH CRN is ideally and uniquely positioned to impact the growing public health significance of NASH that can only be addressed via a larger research consortium. The primary objective of the NASH CRN is to perform clinical research on NASH and NAFLD in adults and children. A closely linked and high priority secondary objective is to conduct translational research in NASH and NAFLD focusing on the pathogenesis that will provide the basis for understanding the natural history and developing means of better diagnosis, treatment, and clinical management. In the next phase of the NASH CRN, the adult and pediatric therapeutic trials initiated during the previous funding cycle will be completed, and new therapeutic trials, including phase 2a proof of mechanism and phase 2b clinical trials will be initiated, to develop evidence-based treatment options that are safe, effective, simple, and inexpensive. The longitudinal cohort of adults and children with NAFLD will be extended, which will prospectively define the natural history of the disease, the cardiovascular and metabolic risk factors, and will aid in biomarker discovery and validation, and development and validation of non-invasive techniques to evaluate and identify those with NASH/NAFLD, who will respond, and how quickly the disease is progressing. Duke University will provide the NASH CRN with expert basic science and clinical-translational expertise in NAFLD, the ability to readily recruit adults and children into prospective clinical trials, and perform ancillary studies to fill existin gaps in knowledge. Duke University and our pediatric sites, Ann & Robert H. Lurie Children's Hospital/JHU, are eager to continue our collaboration with NIDDK program staff, the Data Coordinating Center, private sector partner and other clinical centers to complete the important research objective of the NASH CRN. The NASH CRN is poised to continue its major impact on the field and directly advance the mission of the National Institutes of Health to improve the health of the public.