Collaborative Clinical Research on Non Alcoholic Stratohepatitis

描述（由申请人提供）： NASH临床研究网络（NASH CRN）成立于2002年，旨在开展与儿童和成人的临床特征，风险因素，发病机制，自然史和治疗相关的研究。NASH CRN的初始资助期在建立和实施网络范围的协议以及许多辅助和试点研究方面非常富有成效。受其成功的鼓舞，NIDDK发布了RFA-DK- 08-505，其目标是将NASH CRN再延续五年。NASH CRN在下一个资助期内的目标是：（a）成功完成初始资助期内启动的三项全网络研究。它们是成人和儿童NAFLD的观察性纵向研究（NAFLD数据库研究，n= 1，215）、非糖尿病成人NASH的随机双风对照治疗研究（PIVENS，n=247）和儿童NAFLD的随机双盲对照治疗研究（TONIC，n=173）。NAFLD数据库研究可能会进行修改，以满足下一个资助期内的其他目标。这些修改可能包括延长入组患者的随访时间，考虑在无肝硬化的患者亚组中进行随访肝活检，以更好地评估组织学自然史，并招募精心选择的无NAFLD的对照组（B），以成功完成在初始资助期间开始的大量辅助研究和试点研究，（c）在患有NASH的成人和儿童中进行额外的治疗研究，因为目前治疗NASH的选择非常有限，以及（d）根据迄今收集的广泛临床材料和生物样本启动并完成额外的辅助研究。这些研究可能集中于（但不限于）儿童和成人NAFLD和NASH的自然史，疾病严重程度的非侵入性评估（例如，蛋白质组学、脂质组学、临床预测规则）和疾病发病机制（细胞因子分析、全基因组关联研究、组织蛋白质组学）。

DESCRIPTION (provided by applicant): 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=l,215), a randomized double Wind 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 number 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 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 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).