Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials.
Importance: One major advantage of developing large, federally funded networks for clinical research in neurology is the ability to have a trial-ready network that can efficiently conduct scientifically rigorous projects to improve the health of people with neurologic disorders.
Observations: National Institute of Neurological Disorders and Stroke Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT) was established in 2011 and renewed in 2018 with the goal of being an efficient network to test between 5 and 7 promising new agents in phase II clinical trials. A clinical coordinating center, data coordinating center, and 25 sites were competitively chosen. Common infrastructure was developed to accelerate timelines for clinical trials, including central institutional review board (a first for the National Institute of Neurological Disorders and Stroke), master clinical trial agreements, the use of common data elements, and experienced research sites and coordination centers. During the first 7 years, the network exceeded the goal of conducting 5 to 7 studies, with 9 funded. High interest was evident by receipt of 148 initial applications for potential studies in various neurologic disorders. Across the first 8 studies (the ninth study was funded at end of initial funding period), the central institutional review board approved the initial protocol in a mean (SD) of 59 (21) days, and additional sites were added a mean (SD) of 22 (18) days after submission. The median time from central institutional review board approval to first site activation was 47.5 days (mean, 102.1; range, 1-282) and from first site activation to first participant consent was 27 days (mean, 37.5; range, 0-96). The median time for database readiness was 3.5 months (mean, 4.0; range, 0-8) from funding receipt. In the 4 completed studies, enrollment met or exceeded expectations with 96% overall data accuracy across all sites. Nine peer-reviewed manuscripts were published, and 22 oral presentations or posters and 9 invited presentations were given at regional, national, and international meetings.
Conclusions and Relevance: NeuroNEXT initiated 8 studies, successfully enrolled participants at or ahead of schedule, collected high-quality data, published primary results in high-impact journals, and provided mentorship, expert statistical, and trial management support to several new investigators. Partnerships were successfully created between government, academia, industry, foundations, and patient advocacy groups. Clinical trial consortia can efficiently and successfully address a range of important neurologic research and therapeutic questions.
Neurosciences (Brain & Spine)
Cudkowicz, Merit; Chase, Marianne K; Coffey, Christopher S; Ecklund, Dixie J; Thornell, Brenda J; Lungu, Codrin; Mahoney, Katy; Gutmann, Laurie; Shefner, Jeremy M; Staley, Kevin J; Bosch, Michael; Foster, Eric; Long, Jeffrey D; Bayman, Emine O; Torner, James; Yankey, Jon; Peters, Richard; Huff, Trevis; Conwit, Robin A; Shinnar, Shlomo; Patch, Donna; Darras, Basil T; Ellis, Audrey; Packer, Roger J; Marder, Karen S; Chiriboga, Claudia A; Henchcliffe, Claire; Moran, Joyce Ann; Nikolov, Blagovest; Factor, Stewart A; Seeley, Carole; Greenberg, Steven M; Amato, Anthony A; DeGregorio, Sara; Simuni, Tanya; Ward, Tina; Kissel, John T; Kolb, Stephen J; Bartlett, Amy; Quinn, Joseph F; Keith, Kellie; Levine, Steven R; Gilles, Nadege; Coyle, Patricia K; Lamb, Jessica; Wolfe, Gil I; Crumlish, Annemarie; Mejico, Luis; Iqbal, Muhammad Maaz; Bowen, J D; Tongco, Caryl; Nabors, Louis B; Bashir, Khurram; Benge, Melanie; McDonald, Craig M; Henricson, Erik K; Oskarsson, Björn; Dobkin, Bruce H; Canamar, Catherine; Glauser, Tracy A; Woo, Daniel; Molloy, Angela; Clark, Peggy; Vollmer, Timothy L; Stein, Alexander J; Barohn, Richard J; Dimachkie, Mazen M; Le Pichon, Jean-Baptiste; Benatar, Michael G; Steele, Julie; Wechsler, Lawrence; Clemens, Paula R; Amity, Christine; Holloway, Robert G; Annis, Christine; Goldberg, Mark P; Andersen, Mariam; Iannaccone, Susan T; Smith, A Gordon; Singleton, J Robinson; Doudova, Mariana; Haley, E Clarke; Quigg, Mark S; Lowenhaupt, Stephanie; Malow, Beth A; Adkins, Karen; Clifford, David B; Teshome, Mengesha A; and Connolly, Noreen, "Seven-Year Experience From the National Institute of Neurological Disorders and Stroke-Supported Network for Excellence in Neuroscience Clinical Trials." (2020). Articles, Abstracts, and Reports. 2939.