Clinical Core

项目摘要/摘要 临床核心中心提供服务和专业知识，并为囊性纤维化提供强大的培训部分 (Cf)研究。这些服务侧重于监管支持，以及研究设计和实施支持。我们的服务是 为合作者提供本地、国内和国际资源，以进行高级临床试验设计和实施， 生物统计学和流行病学方法以及包括成本效益在内的比较效果研究方法 分析。核心支持华盛顿大学囊性纤维化研究和翻译中心的中心目标 (CFRTC)：使用高效调节器疗法(HEMT)启动以发现囊性疾病的新方面 纤维化(CF)病理生理学，定义临床表现和最佳治疗方法 并对持续存在的疾病方面进行基础和转化性研究 尽管接受了HEMT。此外，CC促进了CFRTC生物医学核心之间的合作，以增强 通过共享扩展的人类和细菌的生存和发展进行翻译研究 链接到临床数据库的存储库，支持改进的分析和临床结果的开发 措施。我们处于有利地位，通过药物开发继续推动新疗法的发展 正在筹备和过渡到主要的CF研究，重点是提高我们对疾病的了解 先进疗法的背景--特别是HEMT，将永远改变CF的临床格局。

PROJECT SUMMARY/ABSTRACT The Clinical Core provides both services and expertise and has a strong training component for cystic fibrosis (CF) research. The services focus on regulatory support, and study design and conduct support. We serve as a local, National and International resource to collaborators for advanced clinical trial design and conduct, biostatistical and epidemiologic methods and comparative effect research methods including cost-effectiveness analysis. The Core supports the central aims of the UW Cystic Fibrosis Research and Translation Center (CFRTC): to use highly effective modulator therapy (HEMT) initiation to discover novel aspects of cystic fibrosis (CF) pathophysiology, define the clinical manifestations and best treatment approaches for the “new” disease that CF will become, and perform basic and translational research on disease aspects that persist despite HEMT. Further, the CC fosters collaboration across the CFRTC biomedical cores to enhance translational research through the sharing of existing and development of expanded human and bacterial repositories linked to clinical databases that enable development of improved assays and clinical outcome measures. We are well positioned to continue to advance novel therapeutics through the drug development pipeline and transition to major studies in CF that focus on improving our understanding of the disease in the setting of advancing therapeutics – specifically HEMT that will forever change the clinical landscape in CF.