Clinical Research Network in Nonalcoholic Steatohepatitis

非酒精性脂肪性肝炎临床研究网络

• 批准号: 9000535
• 负责人: KRIS KOWDLEY
• 金额: $ 4.18万
• 依托单位: SWEDISH MEDICAL CENTER, FIRST HILL
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-01-27 至 2019-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9000535
• 关键词: Address; Adolescent; Adult; Affect; Ancillary Study; Biological Markers; Cardiovascular system; Child; Childhood; Clinical; Clinical Management; Clinical Research; Clinical Trials; Collaborations; Cysteamine; Data; Databases; Development; Diagnosis; Diagnostic; Disease; Disease Progression; Enrollment; Evidence based treatment; Fatty Liver; Feasibility Studies; Funding; Future; Genetic; Health; Image; Incidence; International; Iron; Laboratory Study; Lead; Link; Liver; Liver diseases; Malignant Neoplasms; Malignant neoplasm of liver; Manuscripts; Medical center; Metabolic; Mission; Modeling; Natural History; North America; Outcome; Pathogenesis; Patient risk; Phase; Pilot Projects; Play; Population; Population Study; Positioning Attribute; Proteomics; Public Health; Randomized; Research; Research Personnel; Risk; Risk Factors; Role; Site; Staging; Techniques; Therapeutic; Therapeutic Trials; Translating; Translational Research; United States; United States National Institutes of Health; Validation; Virginia; adverse outcome; base; clinical application; clinical material; clinical practice; clinical research site; cohort; cost; disease natural history; follow-up; improved; longitudinal database; meetings; metabolomics; mortality; non-alcoholic fatty liver; nonalcoholic steatohepatitis; novel therapeutics; public-private partnership; response; success; translational study

DESCRIPTION (provided by applicant): This application is for the Continuation of the Virginia Mason Medical Center (VMMC) Clinical Site and its Subsites of the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN). 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 large 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. VMMC has played a leading role in both the clinical and translational research success of the NASH CRN since the inception. VMMC was a leading enrolling site in both the PIVENS and FLINT trials. VMMC currently has 5 ongoing NASH CRN approved ancillary studies, including AS40, "The Role of Iron Pathogenesis of NAFLD" (R01DK087696), which has made major contributions toward understanding the effect of iron in NAFLD. VMMC investigators have contributed 20 presentations at national and international scientific meetings and coauthored 14 manuscripts on behalf 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.

描述（由申请人提供）：本申请适用于弗吉尼亚梅森医疗中心 (VMMC) 临床站点及其非酒精性脂肪性肝炎临床研究网络 (NASH CRN) 子站点的延续。非酒精性脂肪肝 (NAFLD) 影响着北美三分之一的成年人和五名儿童，并且是美国日益严重的公共卫生问题。 NAFLD，特别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，以及肝脏、心血管和癌症相关的死亡率，导致健康负担和费用大幅增加。 NASH CRN 具有理想且独特的地位，可以影响 NASH 日益增长的公共卫生重要性，而这一问题只能通过大型研究联盟来解决。 NASH CRN 的主要目标是对成人和儿童的 NASH 和 NAFLD 进行临床研究。一个密切相关且高度优先的次要目标是开展 NASH 和 NAFLD 的转化研究，重点关注发病机制，这将为了解自然史和开发更好的诊断、治疗和临床管理手段提供基础。 NASH CRN下一阶段将完成上一资助周期启动的成人和儿童治疗试验，并启动新的治疗试验，包括2a期机制证明和2b期临床试验，以开发循证医学安全、有效、简单且廉价的治疗方案。患有 NAFLD 的成人和儿童的纵向队列将得到扩展，这将前瞻性地定义疾病的自然史、心血管和代谢危险因素，并将有助于生物标志物的发现和验证，以及非侵入性技术的开发和验证。评估和识别 NASH/NAFLD 患者、谁会做出反应以及疾病进展的速度。自 NASH CRN 成立以来，VMMC 在临床和转化研究的成功中发挥了主导作用。 VMMC 是 PIVENS 和 FLINT 试验中领先的招募网站。 VMMC 目前有 5 项正在进行的 NASH CRN 批准的辅助研究，包括 AS40，“铁在 NAFLD 发病机制中的作用”(R01DK087696)，该研究为了解铁在 NAFLD 中的作用做出了重大贡献。 VMMC 研究人员在国内和国际科学会议上发表了 20 篇演讲，并代表 NASH CRN 共同撰写了 14 篇手稿。 NASH CRN 准备继续在该领域发挥重大影响，并直接推进美国国立卫生研究院改善公众健康的使命。