Clinical Research Sites for the Network of Excellence in Neuroscience Clinical Tr

描述（由申请人提供）：迫切需要将新的临床前数据快速转化为各种神经系统疾病患者的新治疗方法。尽管基础科学知识呈指数级增长，但将新发现转化为治疗方法的研究却非常有限。有多种原因导致在250个经过临床前测试的分子中，只有不到1个最终被批准用于临床使用;一个重要因素是难以招募患有低患病率疾病的受试者。该应用程序是为了响应NINDS RFA而开发的，以作为临床研究中心参与神经科学临床试验卓越网络（NEXT）。西北大学范伯格医学院（NUFSM）的神经病学临床试验中心（NCTC）是一个协调三个主要西北大学附属临床设施的教师和资源的协作努力：西北纪念医院（NMH），儿童纪念医院（CMH）和芝加哥康复研究所（RIC）。NCTC的教师是由高技能的临床研究人员与专业知识，在所有神经亚专科和访问大型 患者基础为患有各种神经系统疾病的多样化且特征明确的成人和儿童人群。NCTC是唯一有资格参与NEXT并为网络临床试验和生物标志物验证研究的开发和成功完成做出贡献的机构。主要人员在各自的子专业中具有丰富的临床和转化研究经验;在主要研究项目中担任领导角色（包括NINDS资助的项目）;尖端合作项目的跟踪记录;接触大量不同的神经系统疾病患者;以及成功招募患者和对照受试者参加由NIH、私人基金会和行业来源赞助的研究和临床试验。我们的西北大学临床和转化科学研究所（NUCATS）的基础设施由NIH资助的临床和转化科学奖（CTSA）和我们的部门NCTC支持，将确保我们中心及时招募受试者。NCTC还拥有大量具有脊髓性肌萎缩症（SMA）研究专业知识的研究人员，并可接触SMA儿童和成人人群。国家反恐中心拥有支持NEXT项目所需的所有资源和设备。此外，西北大学（NU）在未来NEXT项目的翻译研究的设计和执行方面拥有丰富的专业知识。参与NEXT将使NCTC能够为成人和儿科人群的多个亚专业的神经学临床试验提供专业知识和研究主题，巩固跨学科的努力，并为初级研究人员提供培训，使他们能够发展独立的研究事业。

DESCRIPTION (provided by applicant): There is a critical need for rapid translation of novel preclinical data into new treatments for patients with a variety of neurological diseases. Despite exponential growth of basic science knowledge, there has been only limited translation of new findings into therapeutics. There are multiple reasons that fewer than 1 in 250 molecules that undergo preclinical testing are ultimately approved for clinical use; one important factor is the difficulty in recruiting subjects who have diseases of low prevalence. This application has been developed in response to the NINDS RFA to participate as a Clinical Site in the Network for Excellence in Neuroscience Clinical Trials (NEXT). The Neurology Clinical Trials Center (NCTC) at Northwestern University's Feinberg School of Medicine (NUFSM) is a collaborative effort that coordinates faculty and resources of three major Northwestern-University-affiliated clinical facilities: Northwestern Memorial Hospital (NMH), Children's Memorial Hospital (CMH) and the Rehabilitation Institute of Chicago (RIC). The NCTC faculty is comprised of highly skilled clinician investigators with expertise across all neurological subspecialties and access to a large patient base of diverse and well-characterized adult and pediatric populations with a variety of neurological diseases. The NCTC is uniquely qualified to participate in NEXT and to contribute to the development and successful completion of the network clinical trials and biomarker validation studies. The key personnel have extensive experience in clinical and translational research in their respective subspecialties; leadership roles in major research programs (including NINDS-funded projects); track records of cutting-edge collaborative projects; access to large and diverse populations of patients with neurological conditions; and success in enrolling patients and control subjects in research studies and clinical trials sponsored by NIH, private foundations and industry sources. The established infrastructures of our Northwestern University Clinical and Translational Sciences Institute (NUCATS) supported by an NIH-funded Clinical and Translational Science Award (CTSA) and our departmental NCTC will assure timely subject recruitment by our Center. The NCTC also has a critical mass of investigators with expertise in spinal muscular atrophy (SMA) research and access to SMA pediatric and adult populations. The NCTC has all of the required resources and equipment necessary to support NEXT projects. In addition, there is substantial expertise at Northwestern University (NU) in the design and execution of translational studies for future NEXT projects. Participation in NEXT will enable the NCTC to contribute expertise and research subjects to neurological clinical trials across multiple subspecialties in adult and pediatric populations, consolidate efforts across disciplines, and provide training for junior investigators to enable them to develop independent research careers.