Kennedy Krieger Institute - Johns Hopkins University NeuroNEXT Site

肯尼迪克里格研究所 - 约翰霍普金斯大学 NeuroNEXT 网站

• 批准号: 10744858
• 负责人: Erika Augustine
• 金额: $ 46.22万
• 依托单位: HUGO W. MOSER RES INST KENNEDY KRIEGER
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-07-15 至 2028-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10744858
• 关键词: Acceleration; Address; Adult; Affect; Agreement; Autoimmune encephalitis; Brain Injuries; Caring; Child; Childhood; Clinical; Clinical Research; Clinical Trials; Clinical and Translational Science Awards; Collaborations; Communities; Complex; Conduct Clinical Trials; Consultations; Data Coordinating Center; Dedications; Development; Disease; Educational Activities; Enrollment; Ensure; Environment; Epilepsy; Equity; Ethnic Origin; Failure; Foundations; Funding; Gene Targeting; Genetic; Geographic Locations; Goals; Huntington Disease; Individual; Infrastructure; Institution; Institutional Review Boards; Intervention; Leadership; Learning; Longevity; Mentors; Mentorship; Migraine; Mission; Multi-Institutional Clinical Trial; Multicenter Trials; Musculoskeletal System; Myopathy; National Center for Advancing Translational Sciences; Neurodevelopmental Disorder; Neurologic; Neurologist; Neurology; Neurosciences; Participant; Patient Representative; Patients; Persons; Phase; Population; Positioning Attribute; Process; Productivity; Race; Rare Diseases; Recommendation; Recording of previous events; Research; Research Personnel; Resources; Sampling; Site; Spinal Injuries; Talents; Therapeutic Intervention; Time; Training; Training Programs; Translational Research; Universities; Voice; Workforce Development; career; clinical development; community based participatory research; community engagement; design; diversity and equity; diversity and inclusion; early phase clinical trial; experience; improved; innovation; interdisciplinary approach; medical schools; member; multidisciplinary; nervous system disorder; neurosurgery; next generation; novel therapeutics; operation; patient population; programs; rare condition; recruit; socioeconomics; success; targeted treatment; therapy development

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.

患有神经系统疾病的儿童和成人往往会经历严重的终身疾病后果。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，对于几种情况，尚未制定有意义的治疗干预措施。创建神经科学临床试验卓越网络（NeuroNEXT）的目的是减少成功进行神经系统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。同样重要的是嵌入NeuroNEXT的使命，以确保下一代神经病学临床试验者接受足够的培训和指导，以扩大设计和进行这些试验的能力。一份咨询性报告于2022年对NeuroNEXT进行了评估，并提出了额外的建议，包括优先考虑儿科人群的试验，将患者的声音纳入试验的开发和加强社区参与，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研究人员队伍的发展。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内广泛的神经病学试验，包括基因靶向治疗和罕见疾病干预; 2）通过使用临床和转化科学奖资源并与NeuroNEXT临床和数据协调中心密切合作，确保试验进行的效率和质量; 3）为NeuroNEXT研究员实施严格的培训计划;以及4）在所有现场活动中嵌入多样性和包容性概念的参与方法，从代表性领导团队开始。我们将通过网络广泛分享我们所学到的东西。我们提供了一个非常有才华和多样化的多学科小组的临床医生和其他研究团队成员，在指导，一个大的和有代表性的患者群体，以及广泛的机构和区域资源进行临床试验的卓越记录。作为一个致力于和合议的领导小组，我们相信KKI-JHU网站将为NeuroNEXT增加独特和巨大的价值，确保NeuroNEXT试验的快速成功和扩大有才华的神经病学试验专家库。