Kennedy Krieger Institute - Johns Hopkins University NeuroNEXT Site

患有神经系统疾病的儿童和成人往往会经历严重的终身后果。严格的临床试验在治疗神经系统疾病方面取得了实质性进展。然而，在一些情况下，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）的创建目标是减少成功进行神经疾病早期临床试验的障碍。该网络取得了进展，不仅进行了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总试验协议，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病学临床试验人员得到充分的培训和指导，以扩大设计和开展这些试验的能力。一个咨询工作组于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的试验，在试验开发中整合患者的声音，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，并进一步促进临床试验研究者队伍的发展。我们的肯尼迪克里格研究所和约翰霍普金斯大学（KKI-JHU）团队渴望通过解决以下几个目标成为旗舰NeuroNEXT网站：1)加强KKI-JHU NeuroNEXT网站基础设施的整合，以支持整个生命周期的广泛神经学试验，包括基因靶向治疗和罕见疾病干预；2)利用临床和转化科学奖资源，与NeuroNEXT临床和数据协调中心密切合作，确保试验进行的效率和质量；3)为NeuroNEXT研究员实施严格的培训计划；4)从具有代表性的领导团队开始，在所有现场活动中嵌入多样性和包容性概念的参与方法。我们将与网络广泛分享我们所学到的知识。我们拥有一支才华横溢、多元化的多学科临床医生团队和其他调查团队成员，在指导方面有着卓越的记录，拥有庞大且具有代表性的患者群体，以及开展临床试验的广泛机构和区域资源。作为一个忠诚的大学领导团队，我们相信KKI-JHU网站将为NeuroNEXT增加独特而巨大的价值，确保NeuroNEXT试验的快速成功和神经病学试验人才的扩大。

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.