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

罗彻斯特大学医学中心及其神经科学研究人员为神经系统疾病的实验治疗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，罗切斯特大学网站（UR-NEXT）参与了11项资助研究中的9项，在前两个奖励期间表现出更好的表现，并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站点的独特定位是为罕见病研究和基因和基因靶向治疗试验的实施做出贡献，并培养早期研究人员成为该领域的领导者。我们有51位潜在的合作研究者，其中包括25位罕见病研究或基因治疗临床试验方面的专家，以及17位我们NINDS T32博士后培训项目（神经疾病实验治疗学）的导师，该项目目前已连续获得资助32年。这些研究人员中的许多人领导着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。作为开展高效、公平、高度完整的成人和儿童神经系统疾病NeuroNEXT临床试验的示范性临床试验场所，我们将继续提高其表现，并通过以下方式加强我们的资质和成就：1)扩大我们在实验治疗方面具有特殊专业知识的杰出合作研究者队伍，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，包括与可信赖的社区成员建立真实的关系，定制和改进我们的招聘和保留方法，以及我们临床试验的多样性；3)通过部署“研究启动包”、社区知情招募和保留活动问卷，以及对每个NeuroNEXT临床试验进行研究后招募策略分析，加强我们的绩效改进方法；4)将早期研究人员纳入我们的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赞助的职业发展奖项；5)在罕见病研究、基因治疗试验和数字工具的整合方面发挥领导作用，同时在NeuroNEXT、NINDS和其他领域开展合作并传播我们的成功。UR-NEXT将是包容的、严谨的研究人员、团队成员、跨界者、流程创新者、熟练的沟通者和系统思考者，以公平地改善受神经疾病影响的儿童、成人和家庭的转化过程和生活。

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.