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Principal Investigator: ROBERT G. HOLLOWAY
Organization: UNIVERSITY OF ROCHESTER
Fiscal Year: 2024
Award: $410,795
Funding agency: National Institute of Neurological Disorders and Stroke
The University of Rochester Medical Center and its neuroscience investigators have provided over four decades of leadership and meaningful contributions to experimental therapeutics for neurological disease. Since the inception of the Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) in 2011, the University of Rochester site (UR-NEXT) has contributed to its success by participating in 9 of 11 funded studies, by showing improved performance over the two prior award periods, and by sharing our expertise throughout the Network. The UR-NEXT site is uniquely positioned to contribute to rare disease research and the implementation of gene and gene-targeted therapy trials, and to train early-stage investigators to be leaders in the field. We have 51 potential co-investigators, including 25 experts in rare disease research or gene-therapy clinical trials, and 17 who are mentors within our NINDS T32 post-doctoral training program, Experimental Therapeutics of Neurological Disease, now in its 32nd year of continuous funding. Many of these investigators lead rare disease clinical trial readiness and enabling research programs with regional, national, and international reach. UR-NEXT will continue to improve its performance as an exemplary clinical trial site that conducts efficient, equitable, and high-integrity NeuroNEXT clinical trials for adult and child neurological diseases, and bolster our qualifications and achievement by: 1) expanding our cadre of exceptional co-investigators with special expertise in experimental therapeutics and to foster their successful contributions to NeuroNEXT and the Gene Therapy Consortium; 2) leading a comprehensive effort of community and multistakeholder engagement, including building authentic relationships with trusted community members, to customize and improve our approach to recruitment and retention, and the diversity in our clinical trials; 3) strengthening our approach to performance improvement by deploying “Study Start-Up Bundles”, a Community-Informed Recruitment and Retention Activity Questionnaire, and by performing a post-study Recruitment Strategy Analysis for each NeuroNEXT clinical trial; 4) integrating early-stage investigators into the career development and training opportunities within our experimental therapeutics training program to ensure their successful transition to an NIH/NINDS-sponsored career development award; and 5) providing leadership in rare disease research, gene therapy trials, and the incorporation of digital tools while collaborating and disseminating our successes across NeuroNEXT, NINDS, and beyond. UR-NEXT will be inclusive, rigorous researchers, team players, boundary crossers, process innovators, skilled communicators, and system thinkers to equitably improve the translational process and the lives of children, adults, and families affected by neurological disease.
Terms: <0-11 years old><21+ years old><Achievement><Achievement Attainment><Adult><Adult Human><Affect><Area><Award><Career Development Awards><Career Development Awards and Programs><Career Development Programs K-Series><Child><Child Health><Child Youth><Children (0-21)><Clinical Sciences><Clinical Trials><Clinical Trials Network><Collaborations><Communities><DNA Therapy><Dentistry><Ensure><Equity><Experimental Therapies><Family><Fortification><Fostering><Funding><Future><Gene Transfer Clinical><Gene therapy trial><Genes><Genetic Intervention><Goals><Grant><IDDRC><IDDRP><Intellectual and Developmental Disabilities Research Centers><International><Investigational Therapies><Investigational Treatments><Investigators><K-Awards><K-Series Research Career Programs><Lead><Leadership><Letters><MRDD Research Center><MRDDRC><Medical center><Mental Retardation and Developmental Disabilities Research Centers><Mentors><NICHD><NIH><NINDS><National Institute of Child Health and Human Development><National Institute of Children's Health and Human Development><National Institute of Neurological Diseases and Stroke><National Institute of Neurological Disorders and Stroke><National Institutes of Health><Nervous System Diseases><Nervous System Disorder><Neurologic Disorders><Neurological Disorders><Neurosciences><Orphan Disease><Pb element><Pediatric Neurology><Performance><Position><Positioning Attribute><Process><Productivity><Qualifying><Questionnaires><Rare Diseases><Rare Disorder><Research><Research Career Program><Research Personnel><Research Resources><Researchers><Resources><Site><Specialist><System><Training><Training Programs><Translation Process><Translational Research><Translational Science><Translations><Trust><United States National Institutes of Health><Universities><Work><adulthood><anti-racism><anti-racist><career development><child neurology><clinical research site><clinical site><clinical trial readiness><community engagement><digital tool><engagement with communities><experience><experimental therapeutic agents><experimental therapeutics><gene repair therapy><gene therapy><gene therapy clinical trial><gene transfer trial><gene-based therapy><gene-based treatment><gene-directed therapy><gene-targeted therapy><gene-targeted treatment><genetic therapy><genomic therapy><heavy metal Pb><heavy metal lead><improved><innovate><innovation><innovative><kids><medical college><medical schools><member><neurological disease><orphan disorder><post-doctoral training><postdoctoral training><programs><recruit><response><school of medicine><skills><success><targeted drug trials><targeted pharmaceutical trials><targeted therapy trials><targeted treatment trials><training opportunity><translation><translation research><translational investigation><youngster>