Continuation of the Nonalcoholic Steatohepatitis Clinical Research Network

非酒精性脂肪性肝炎临床研究网络的延续

基本信息

批准号：
8600763
负责人：
NORAH A. TERRAULT
金额：
$ 3.03万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
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：
2002-06-15 至 2014-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8600763
关键词：
Address Adult Affect American Ancillary Study Area Attention Biological Markers Biopsy Burn injury Cardiac Cardiovascular Diseases Cardiovascular system Caring Child Cirrhosis Clinic Clinical Clinical Research Clinical Trials Clinical Trials Database Control Groups Data Databases Development Diabetes Mellitus Diagnosis Diffusion Disease Disease Progression Double-Blind Method Elements Enrollment Evaluation Fatty Liver Feasibility Studies Funding Future Genetic Goals Health Histology Image Incidence Individual Investigation Length Liver Liver Fibrosis Liver diseases Longitudinal Studies Magnetic Resonance Imaging Mass Spectrum Analysis Metabolic Methods Modification Monitor Myocardial Myocardium National Institute of Diabetes and Digestive and Kidney Diseases Natural History North America Pathogenesis Patients Performance Phase Phenotype Pilot Projects Prevention Proteomics Protocols documentation Protons Publications Randomized Randomized Clinical Trials Reporting Research Risk Risk Factors Sampling Serum Severities Severity of illness Site Specificity Staging Systems Biology Techniques Testing Therapeutic Studies Therapy Clinical Trials Tissues Triglycerides Underrepresented Minority United States United States Dept. of Health and Human Services United States National Institutes of Health Work bariatric surgery base chronic liver disease clinical material cohort cytokine disease natural history effective therapy follow-up genome wide association study insulin sensitizing drugs lipid disorder liver biopsy meetings metabolomics non-alcoholic fatty liver non-diabetic nonalcoholic steatohepatitis novel prospective public health relevance sobriety success tool

项目摘要

DESCRIPTION (provided by applicant): Nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) are important causes of chronic liver disease in the United States. The NASH Clinical Research Network (NASH CRN) was established in 2002 to conduct research related to its clinical features, risk factors, pathogenesis, natural history and treatment in children and adults. The initial funding period of the NASH CRN has been very productive in establishing and conducting network-wide protocols and numerous ancillary and pilot studies. Encouraged by its success, the NIDDK has issued RFA-DK-08-505 whose objective is to continue the NASH CRN for additional five years. The objectives of the NASH CRN during the next funding period are (a) to successfully complete three network-wide studies initiated during the initial funding period. They are an observational longitudinal study of NAFLD in adults and children (NAFLD Database study, n=1,215), a randomized double blind controlled therapeutic study of NASH in non-diabetic adults (PIVENS, n=247), and a randomized double blind controlled therapeutic study of NAFLD in children (TONIC, n=173). The NAFLD Database study may be amended to meet additional goals during the next funding period. These modifications may include extending the length of follow-up of those enrolled, consideration of a follow-up liver biopsy in a subset of patients without cirrhosis for better assessment of histological natural history and enrolling carefully chosen controls without NAFLD; (b) to successfully complete a large of ancillary studies and pilot studies that have been begun during the initial funding period; (c) to conduct additional therapeutic studies in adults and children with NASH as options currently available to treat NASH are quite limited. Subsets of patients requiring immediate attention include (but not limited) to those with diabetes or cirrhosis and (d) to initiate and complete additional ancillary studies based on extensive clinical material and biosamples collected thus far. These studies may focus on (but are not limited to) natural history of NAFLD and NASH in children and adults, non-invasive assessment of disease severity (e.g., proteomics, lipodimics, clinical prediction rules) and disease pathogenesis (cytokine analyses, genome-wide association studies, tissue proteomics). Two new ancillary studies proposed by the UCSF Clinical Center aim to: a) Develop a non-invasive alternative to liver biopsy for the evaluation and monitoring of hepatic fibrosis in NAFLD CRN patients using novel MRI imaging and MR spectroscopic techniques, and b) Elucidate the impact of NAFLD on cardiac lipotoxicity, cardiac energetics, and myocardial function in NASH CRN patients using cardiac MRI and proton mass spectroscopy.

描述（由申请人提供）：非酒精性脂肪肝病（NAFLD）和非酒精性脂肪性肝炎（NASH）是美国慢性肝病的重要原因。 NASH临床研究网络（NASH CRN）于2002年成立，旨在进行与其临床特征，危险因素，发病机理，自然病史和儿童和成人治疗有关的研究。 NASH CRN的最初资助期在建立和进行网络范围的方案以及众多辅助和试点研究方面非常有生产力。在成功的鼓励下，NIDDK发行了RFA-DK-08-505，其目标是继续纳什CRN五年。在下一个资金期间，NASH CRN的目标是（a）成功完成在初始资金期间启动的三项网络范围的研究。它们是对成人和儿童NAFLD的观察纵向研究（NAFLD数据库研究，n = 1,215），这是一项随机的双盲盲对照治疗研究，对非糖尿病成年人的NASH（Pivens，n = 247），以及NAFLD随机的双盲疗法研究，以及Nafld的NAFLD在儿童中（Tonic n = 173）。 NAFLD数据库研究可以修改以在下一个资金期间实现其他目标。这些修改可能包括延长入学者的随访时间，考虑到没有肝硬化的一部分患者的随访活检，以更好地评估组织学自然病史并招募没有NAFLD的精心选择的对照；（b）成功完成在初始资金期间开始的大量辅助研究和试点研究；（c）在成人和纳什儿童中作为目前可以治疗纳什的选项进行其他治疗研究非常有限。需要立即注意的患者子集包括（但不限于）患有糖尿病或肝硬化的患者，以及（d）基于迄今为止收集的广泛临床材料和生物样本，启动并完成其他辅助研究。这些研究可能集中在儿童和成人中NAFLD和NASH的自然历史上，对疾病严重程度（例如蛋白质组学，脂质症，脂质症，临床预测规则）和疾病发病机理（细胞因子分析，全基因组关联研究，组织蛋白质组学）的非侵入性评估。 Two new ancillary studies proposed by the UCSF Clinical Center aim to: a) Develop a non-invasive alternative to liver biopsy for the evaluation and monitoring of hepatic fibrosis in NAFLD CRN patients using novel MRI imaging and MR spectroscopic techniques, and b) Elucidate the impact of NAFLD on cardiac lipotoxicity, cardiac energetics, and myocardial function in NASH CRN patients using cardiac MRI和质子质谱。