Clinical Research Site for the Network of Excellence in Neuroscience Clin. Trials

描述(申请人提供)：尽管最近取得了进展，但神经疾病仍然是一个主要的公共卫生问题，不仅在美国，而且在世界范围内都是重大发病率和死亡率的来源。随着神经科学中新知识的爆炸性增长，整体神经疾病负担大幅减少的可能性存在，但将新的实验室发现转化为新的人类治疗方法的工作一直滞后，部分原因是在组织、资助和执行必要的人类临床试验以证明新治疗方法的有效性方面效率低下。神经科学临床试验卓越网络(NEXT)旨在帮助加快这一过程的至少一部分，特别是进行早期多中心临床试验，以确定安全性和剂量信息，或验证生物标记物和临床结果，为中后期疗效试验做准备。该应用程序建议将弗吉尼亚大学卫生系统作为下一个网络的临床站点。该提案描述了可用于指导的多学科临床神经学研究人员的非凡经验和专业知识 Next临床试验；一个现有的临床推广计划，该计划将能够接触到Rura少数民族和医疗服务不足的患者群体；一个全州神经学实践的推广计划，该计划将在弗吉尼亚州联邦范围内最大限度地招募试验；以及研究基础设施和领导计划，以在弗吉尼亚大学成功实施Next临床试验。 公共卫生相关性：该提案描述了如何组织弗吉尼亚大学的研究人员与其他人合作，在患者中进行重要的新研究，以帮助为患有脑、脊髓、肌肉和神经疾病的人找到新的治疗方法。这项研究可能会对公共卫生产生重大影响。

DESCRIPTION (provided by applicant): Despite recent advances, neurological diseases continue to be a major public health problem and are the source of significant morbidity and mortality not only in the United States, but world-wide. With the explosion of new knowledge in the neurosciences, the possibility of dramatic reductions in the overall neurological disease burden exists, but translation of new laboratory findings into new human treatments has lagged partially due to inefficiencies in the organization, funding, and execution of the necessary human clinical trials to prove the efficacy of new therapeutic approaches. The Network for Excellence in Neuroscience Clinical Trials (NEXT) is proposed to help expedite at least part of this process, specifically the conduct of early phase multi-center clinical trials to establish safety and dose information or to validate biomarkers and clinical outcomes in preparation for middle and late phase trials of efficacy. This application proposes the University of Virginia Health System as a Clinical Site for the NEXT network. The proposal describes the extraordinary experience and expertise of a multi-disciplinary cadre of clinical neurological investigators available to conduct NEXT clinical trials, an already existing clinical outreach program that will afford access to rura minority and medically underserved patient populations, an outreach program with statewide neurology practices that will maximize trial recruitment throughout the Commonwealth of Virginia, and the research infrastructure and leadership plan for successfully executing NEXT clinical trials at the University of Virginia. PUBLIC HEALTH RELEVANCE: This proposal describes how investigators at the University of Virginia, in collaboration with others, will be organized to conduct important new research in patients to help find new treatments for people with diseases of the brain, spinal cord, muscle, and nerves. This research will have potentially major public health implications.