Kennedy Krieger Institute - Johns Hopkins University NeuroNEXT Site - PROJECT SUMMARY/ABSTRACT 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 generalizability of trial results, 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 all site activities, beginning with a leadership team with broad and comprehensive experience. We will broadly share what we learn with the network. We offer an extraordinarily talented and multidisciplinary group of clinicians and other investigative team members, a track record of excellence in mentorship, a large 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 e
