Kennedy Krieger Institute - Johns Hopkins University NeuroNEXT Site

患有神经系统疾病的儿童和成人通常会经历他们的疾病带来的严重的终生后果。严格的临床试验在治疗神经疾病患者方面取得了实质性进展。然而，在几种情况下，有意义的治疗干预措施的发展尚未实现。神经科学临床试验卓越网络(NeuroNEXT)的创建目的是减少神经系统疾病早期临床试验成功的障碍。该网络取得了进展，不仅通过按时或提前完成登记的试验，而且通过使用中央机构审查委员会和主要试验协议成功地加快了试验的启动。同样重要的是，NeuroNEXT的使命是确保下一代神经学临床试验人员得到足够的培训和指导，以扩大设计和实施这些试验的能力。一个咨询工作组在2022年对NeuroNEXT进行了评估，并提出了其他建议，包括优先考虑儿科人群中的试验，将患者的声音纳入试验的开发并加强社区参与，提高试验参与者和研究团队成员的种族和民族代表性，以及进一步促进临床试验调查员队伍的发展。我们的肯尼迪-克里格研究所和约翰霍普金斯大学(KKI-JHU)团队渴望通过实现以下几个目标成为NeuroNEXT的旗舰网站：1)加强KKI-JHU NeuroNEXT网站基础设施的整合，以支持全生命周期内以神经病学为重点的广泛试验，包括基因靶向治疗和罕见疾病干预；2)利用临床和翻译科学奖资源，并与NeuroNEXT临床和数据协调中心密切合作，确保试验的效率和质量；3)对NeuroNEXT研究员实施严格的培训计划；以及4)从具有代表性的领导团队开始，在所有网站活动中嵌入多样性和包容性的概念。我们将广泛分享我们在网络中学到的东西。我们提供了一支才华横溢且多元化的临床医生和其他研究团队成员组成的多学科团队，在指导方面的卓越记录，大量具有代表性的患者群体，以及进行临床试验的广泛机构和地区资源。作为一个致力于合作的领导者小组，我们相信KKI-JHU网站将为NeuroNEXT增加独特和巨大的价值，确保NeuroNEXT试验的快速成功和神经学试验人才库的扩大。

Children and adults with neurological conditions often experience significant, lifelong consequences of their illness. Rigorous clinical trials have yielded substantial advances in care for people with neurological disorders. For several conditions, however, the development of meaningful therapeutic interventions has not materialized. The Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) was created with a goal of reducing barriers to successful early-phase clinical trials for neurological diseases. The network has made progress, not only by conducting trials with on-time or early enrollment completion, but also by succeeding in expediting trial start-up through use of a central Institutional Review Board and master trial agreements. Equally important is the mission embedded in NeuroNEXT to ensure the next generation of neurology clinical trialists receives adequate training and mentorship to expand the capacity to design and conduct these trials. An advisory workgroup evaluated NeuroNEXT in 2022 and set forth additional recommendations, including prioritizing trials in pediatric populations, integrating the patient voice in the development of trials and strengthening community engagement, improving the racial and ethnic representativeness of trial participants and research team members, and further enabling the development of the clinical trial investigator workforce. Our Kennedy Krieger Institute and Johns Hopkins University (KKI-JHU) team is eager to become a flagship NeuroNEXT site by addressing several aims: 1) bolstering the integration of KKI-JHU NeuroNEXT site infrastructure to support wide-ranging neurology-focused trials across the lifespan, including gene-targeted therapies and rare disease interventions; 2) ensuring the efficiency and quality of trial conduct by using Clinical and Translational Science Award resources and closely working with the NeuroNEXT Clinical and Data Coordinating Centers; 3) implementing a rigorous training program for NeuroNEXT fellows; and 4) embedding an engaged approach to the concepts of diversity and inclusion in all site activities, beginning with a representative leadership team. We will broadly share what we learn with the network. We offer an extraordinarily talented and diverse multidisciplinary group of clinicians and other investigative team members, a track record of excellence in mentorship, a large and representative patient population, and extensive institutional and regional resources for conducting clinical trials. As a committed and collegial group of leaders, we are confident that the KKI-JHU site will add unique and tremendous value to NeuroNEXT, ensuring the rapid success of NeuroNEXT trials and expansion of a talented pool of neurology trialists.