Clinical Coordinating Center for the Network of Excellence in Neuroscience Clinical Trials (NEXT - CCC)

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Project Summary The long-term objective of the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) initiative is to efficiently translate advances in neuroscience into treatments for adults and children with neurological disorders through partnerships with government, academia, private foundations, and industry. Recent discoveries in basic neuroscience have directly led to major breakthroughs in the understanding of disease processes in neurological disorders, many of which impose an enormous burden on individuals, families, and communities. Yet there are several organizational and logistical challenges to testing these new therapies in multicenter clinical trials, including inefficiencies in regulatory and institutional approvals, barriers to recruitment and retention of diverse study participants, and a dearth of individuals trained to be the leaders of multicenter clinical research trials. A central goal of NeuroNEXT (NN) is to facilitate, from initial conception through final analysis, high-quality early phase clinical trials and biomarker multisite studies with clear go/no-go decisions by providing efficient methodological, organizational, statistical, and logistical support. NeuroNEXT will also serve as the vehicle for the conduct of early clinical trials of gene therapy for ultra-rare neurological disorders. If warranted by the nature of the studied population, such as rare diseases, adaptive phase 2-3 trials and platform trials will be used. NeuroNEXT is comprised of 25 clinical sites, a Clinical Coordinating Center (CCC), and a Data Coordinating Center (DCC). Aim 1 is to create a collaborative and accessible CCC and clinical research network with the DCC, to efficiently conduct high-quality Phase 2 clinical trials, biomarker studies, and gene therapy trials in rare and common adult and pediatric neurological disorders. Aim 2 is to expand the pool and diversity of experienced clinical Investigators and research staff who are prepared to be leaders of multicenter clinical research trials. Aim 3 is to form strategic collaborations with key stakeholders in clinical trials, including government organizations, academia, foundations, industry partners, and patient-centered groups to organize high-impact clinical trials through NN for neurological disorders. NeuroNEXT innovations will include establishing a trial network that requires the use of a single institutional review board, a master contract process for all network- funded studies, centralized pharmacy and laboratory testing, a single Data Safety Monitoring Board for safety review, broad sharing of Standard Operating Procedures and knowledge, state-of-the-art early development trial design approaches, and the use of the common data elements for data collection. Applicants who seek to use the network can be novice or experienced trialists, thereby greatly expanding the pool and diversity of experienced clinical investigators trained and prepared to lead multicenter trials. New protocols will be proactively sought through several new outreach approaches. Eleven studies involving partnerships with industry, foundations, academia, and the government were funded in the first 11 years NN. The CCC will expand on these prior achievements to further advance and accelerate therapeutic development for neurological disorders.