Columbia University Medical Center - Weill Cornell Medical Center NEXT Site

描述（由申请人提供）：哥伦比亚大学医学中心(CUMC)-威尔康奈尔医学中心（WCMC） NEXT站点将通过创建共享基础设施来提高由NINDS赞助的临床研究的效率，该基础设施将受益于现有资源，包括两个CTSAs，与行业和基金会合作进行II期探索性试验，其中一些可能是生物标志物信息。这两个神经科学中心在研究方面有着悠久的合作历史；各自的医院合并（纽约长老会医院）；而且他们都有一个地区联盟的CTSAs。我们的目标是：1)在CUMC （hub）和WCMC （spoke）建立一个神经科学临床试验点，该试验点将吸收成人和儿童神经病学、神经外科、基础神经科学和神经流行病学方面的广泛专业知识，并能够在其整个生命周期内进行5至7个二期临床试验或生物标志物验证研究；2)利用CTSA和各中心临床试验办公室的资源，有效地识别和招募不同种族的参与者，并迅速招募和跟踪试验完成；3)培养下一代临床和基础神经科学家成为独立的研究者，他们将设计和开展临床试验，利用新的技术和试验方法；4)在国家层面上参与并贡献NEXT网络。我们已经确定了在NIH，行业和基金会支持的临床试验中有经验的共同研究者，证明了与基金会的伙伴关系，并承诺指导。我们利用各自的社区、门诊护理网络和先进的生物信息学，制定了针对罕见和更常见的神经系统疾病的招募策略。我们的招聘工作将与我们的三管齐下的目标相吻合：1)通过双语（英语和西班牙语）针对疾病的项目对家庭进行教育，2)为倡导者建立一个临床研究学习机构，3)为教师职业发展组织一个多方面的项目。大规模的SMA人群（n=106）和成功的SMA生物标志物研究入组为我们提供了进行预期的NEXT SMA生物标志物验证试验所需的经验。

DESCRIPTION (provided by applicant): The Columbia University Medical Center (CUMC)-Weill Cornell Medical Center (WCMC) NEXT site will increase the efficiency of clinical research sponsored by NINDS through the creation of a shared infrastructure that will benefit from existing resources, including two CTSAs, to conduct Phase II exploratory trials, some of which may be biomarker-informed, in partnership with industry and foundations. The two neuroscience centers have a long history of collaboration in research; their respective hospitals are combined (New York Presbyterian Hospital); and they both have CTSAs linked in a regional consortium. Our aims are: 1) to develop a neuroscience clinical trials site at CUMC (hub) and WCMC (spoke) that draws from extensive expertise in adult and pediatric neurology, neurosurgery, basic neuroscience, and neuroepidemiology and that is capable of conducting five to seven Phase II clinical trials or biomarker validation studies across its lifespan; 2) to leverage resources from the CTSA and Office of Clinical Trials at each center to efficiently identify and recruit ethnically diverse participants who are expeditiously recruited and followed to trial completion; 3) to train the next generation of clinical and basic neuroscientists to become independent investigators who will design and conduct clinical trials, making use of new technologies and trial methodologies; and 4) to participate in, and contribute to, the NEXT network on a national level. We have identified co-investigators who have experience in NIH-, industry- and foundation-supported clinical trials, demonstrated partnership with foundations, and a commitment to mentorship. We have developed a recruitment strategy for rare and more common neurological diseases, making use of our respective communities, ambulatory care networks, and advanced bioinformatics. Our recruitment efforts will dovetail with our three-pronged goal to: 1) educate families through bilingual (English and Spanish) disease-specific programs, 2) develop a clinical-research learning-institute for advocates, and 3) organize a multifaceted program for faculty career-development. The large SMA population (n=106) and demonstrated successful enrollment in the Biomarker for SMA study provides us with the necessary experience to conduct the anticipated NEXT SMA biomarker validation trial.