University of Rochester Clinical Site within the NEXT Network of Clinical Trials

NEXT 临床试验网络内的罗切斯特大学临床站点

• 批准号: 10743143
• 负责人: ROBERT G. HOLLOWAY
• 金额: $ 42.35万
• 依托单位: UNIVERSITY OF ROCHESTER
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-07-01 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10743143
• 关键词: Achievement; Adult; Affect; Area; Award; Child; Child Health; Clinical Sciences; Clinical Trials; Clinical Trials Network; Collaborations; Communities; Dentistry; Ensure; Equity; Family; Fostering; Funding; Future; Gene Targeting; Gene therapy trial; Genes; Goals; Grant; Intellectual and Developmental Disabilities Research Centers; International; Investigational Therapies; K-Series Research Career Programs; Lead; Leadership; Letters; Medical center; Mentors; National Institute of Child Health and Human Development; National Institute of Neurological Disorders and Stroke; Neurosciences; Pediatric Neurology; Performance; Positioning Attribute; Process; Productivity; Qualifying; Questionnaires; Rare Diseases; Research; Research Personnel; Resources; Site; Specialist; System; Training; Training Programs; Translation Process; Translational Research; Translations; Trust; United States National Institutes of Health; Universities; Work; anti-racism; career development; clinical research site; clinical trial readiness; community engagement; digital tool; experience; fortification; gene therapy; gene therapy clinical trial; improved; innovation; medical schools; member; nervous system disorder; post-doctoral training; programs; recruit; response; skills; success; targeted therapy trials; training opportunity

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.

罗切斯特大学医学中心及其神经科学研究人员在神经系统疾病的实验治疗方面提供了四十多年的领导和有意义的贡献。自2011年神经科学临床试验卓越网络（NeuroNEXT）成立以来，罗切斯特大学（UR-NEXT）通过参与11项资助研究中的9项，通过在前两个奖项期间表现出更好的表现，以及通过在整个网络中分享我们的专业知识，为其成功做出了贡献。UR-NEXT网站的独特定位是为罕见疾病研究和基因和基因靶向治疗试验的实施做出贡献，并培训早期研究人员成为该领域的领导者。我们有51名潜在的合作研究者，其中包括25名罕见疾病研究或基因治疗临床试验专家，以及17名NINDS T32博士后培训计划（神经疾病实验治疗学）的导师，目前已连续资助32年。这些研究人员中的许多人领导了罕见病临床试验准备工作，并使研究项目具有区域，国家和国际影响力。UR-NEXT将继续提高其作为一个示范性临床试验网站的表现，为成人和儿童神经系统疾病进行有效，公平和高度完整的NeuroNEXT临床试验，并通过以下方式提高我们的资格和成就：1）扩大我们在实验治疗方面具有特殊专长的杰出合作研究者的队伍，并促进他们对NeuroNEXT和基因治疗联盟的成功贡献; 2）领导社区和多方利益相关者参与的全面努力，包括与值得信赖的社区成员建立真实的关系，以定制和改进我们的招募和保留方法以及我们临床试验的多样性; 3）通过部署“研究启动包”，一个社区知情的招聘和保留活动问卷，并通过对每个NeuroNEXT临床试验进行研究后招募策略分析; 4）将早期研究者纳入我们实验治疗培训计划中的职业发展和培训机会，以确保他们成功过渡到NIH/NINDS赞助的职业发展奖;和5）在罕见疾病研究、基因治疗试验和数字化工具整合方面发挥领导作用，同时在NeuroNEXT、NINDS等领域合作和传播我们的成功。UR-NEXT将是包容性的，严谨的研究人员，团队合作者，跨界者，流程创新者，熟练的沟通者和系统思考者，以公平地改善翻译过程和受神经系统疾病影响的儿童，成人和家庭的生活。

项目成果

Safety and Efficacy of Topiramate in Individuals With Cryptogenic Sensory Peripheral Neuropathy With Metabolic Syndrome: The TopCSPN Randomized Clinical Trial.

托吡酯对伴有代谢综合征的隐源性感觉周围神经病患者的安全性和有效性：TopCSPN 随机临床试验。

• DOI: 10.1001/jamaneurol.2023.3711
• 发表时间: 2023
• 期刊: JAMA neurology
• 影响因子: 29
• 作者: Smith,AGordon;Singleton,JRobinson;Aperghis,Adrienne;Coffey,ChristopherS;Creigh,Peter;Cudkowicz,Merit;Conwit,Robin;Ecklund,Dixie;Fedler,JanelK;Gudjonsdottir,Anna;Hauer,Peter;Herrmann,DavidN;Kearney,Marianne;Kissel,John;Klin
• 通讯作者: Klin