Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) DCC

项目总结/摘要 神经科学临床试验卓越网络（NeuroNEXT）倡议的长期目标是快速有效地将神经科学的进步转化为神经系统疾病的治疗方法。有超过1000种不同的神经系统疾病，其中许多与严重的认知和身体残疾有关。在美国，神经系统疾病影响超过1亿的所有年龄的人，并且它们是死亡和残疾的重要原因。虽然目前对许多这些疾病的治疗选择有限或不存在，但神经科学和神经系统疾病基因疗法的最新令人兴奋的进展使得必须提高有效进行临床试验的能力。NeuroNEXT的一个中心目标是促进从最初的概念到最终分析，高质量的早期临床试验和生物标志物研究，在成人和儿童神经系统疾病中具有明确的去/不去决定。该申请要求为爱荷华州大学提供资金，以继续担任NeuroNEXT的DCC。DCC将继续支持研究设计、数据收集、数据管理、数据共享、项目管理、临床研究中心监查、质量管理、安全性监查以及在网络内进行的试验的所有统计方面。DCC将继续建立在我们强大的，标准化的和可访问的基础设施上，以明确的方式设计，以适应临床试验中自然发生的动态变化的要求，并将继续帮助NeuroNEXT成功地提供更快速的评估神经科学中有前途的治疗方法。具体目标1是与CCC密切合作，继续支持一个合作网络社区，有效地征求和进行高质量的神经系统疾病临床试验。具体目标2是继续为所有网络临床试验提供高质量的IT/开发、数据管理、统计和方案协调支持。具体目标3是扩大有经验的临床研究者、研究人员和生物统计学家的人才库，他们准备成为多中心临床研究试验的领导者。

PROJECT SUMMARY/ABSTRACT The long-term objective of the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) initiative is to rapidly and efficiently translate advances in neuroscience into treatments for neurological disorders. There are over 1000 different neurological disorders, many of which are associated with significant cognitive and physical disabilities. In the United States, neurological diseases affect over 100 million people of all ages, and they are a significant cause of mortality and disability. While therapeutic options are currently limited or non-existent for many of these disorders, recent exciting advances in the neurosciences and in gene therapies for neurological disorders make it imperative to increase the ability to efficiently perform clinical trials. A central goal of NeuroNEXT is to facilitate, from initial conception through final analysis, high-quality early phase clinical trials and biomarker studies with clear go/no-go decisions in adult and pediatric neurological disorders. This application requests funds for the University of Iowa to continue serving as the DCC for NeuroNEXT. The DCC will continue to support study design, data collection, data management, data sharing, project management, clinical site monitoring, quality management, safety monitoring, and all statistical aspects of trials conducted within the network. The DCC will continue to build on our robust, standardized, and accessible infrastructure in a manner that is explicitly designed to accommodate dynamically changing requirements that naturally occur in clinical trials, and will continue to help NeuroNEXT succeed in providing more rapid evaluations of promising treatments in the neurosciences. Specific Aim 1 is to work closely with the CCC to continue to support a collaborative network community that efficiently solicits and conducts high-quality clinical trials for neurological disorders. Specific Aim 2 is to continue to provide high-quality IT/development, data management, statistical, and protocol coordination support for all network clinical trials. Specific Aim 3 is to expand the pool of experienced clinical Investigators, research staff, and biostatisticians who are prepared to be leaders of multicenter clinical research trials.