Columbia University Medical Center - Weill Cornell Medical Center NEXT Site

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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.