The Saint Louis University Component of the NASH Clinical Research Network

描述(申请人提供)：本申请是圣路易斯大学非酒精性脂肪性肝炎临床研究网络(NASH CRN)临床中心的延续。NASH临床研究网络(NASH CRN)自2002年以来一直由NIDDK赞助，并于2009年续展。非酒精性脂肪性肝病(NAFLD)在北美影响着三分之一的成年人和五分之一的儿童，因此在美国是一个新兴的公共卫生问题。非酒精性脂肪肝，尤其是非酒精性脂肪性肝炎(NASH)，可能导致肝硬变和原发性肝癌，导致死亡或肝移植，从而导致健康负担和成本的重大增加。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，只能通过大型研究联盟来解决。NASH CRN的主要目标是在成人和儿童中进行NASH和NAFLD治疗药物的临床试验。一个密切联系和高度优先的次要目标是在NASH和NAFLD中进行转化性研究，重点是发病机制，这将为了解自然历史和开发更好的诊断、预防、治疗和临床管理手段提供基础。在NASH CRN的下一阶段，将完成在前一个资金周期启动的成人和儿童治疗试验，并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。过去十年收集的成人和儿童NAFLD纵向队列将扩大到前瞻性地定义疾病的自然病史、心血管和代谢风险因素，并将有助于生物标记物的发现和验证。这一队列还将促进非侵入性技术的开发和验证，以识别NASH/NAFLD患者，预测谁将对治疗有效，并确定影响疾病进展的因素。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) clinical center at Saint Louis University. The NASH Clinical Research Network (NASH CRN) has been sponsored by the NIDDK since 2002 with a renewal in 2009. Nonalcoholic fatty liver disease (NAFLD) affects one out of three adults and one in five children in North America and is thus an emerging public health issue in the United States. NAFLD, and especially nonalcoholic steatohepatitis (NASH), may lead to cirrhosis and primary liver cancer resulting in death or liver transplant thus leading to 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 large research consortium. The primary objective of the NASH CRN is to perform clinical trials of therapeutic agents for 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, prevention, 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 collected over the past decade will be extended to prospectively define the natural history of the disease, the cardiovascular and metabolic risk factors, and will aid in biomarker discovery and validation. This cohort will also facilitate the development and validation of non-invasive techniques to identify those with NASH/NAFLD, predict who will respond to treatments, and identify factors affecting disease progression. 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.