Tanya Simuni, Brian Fiske, Kalpana Merchant, Christopher S Coffey, Elizabeth Klingner, Chelsea Caspell-Garcia, David-Erick Lafontant, Helen Matthews, Richard K Wyse, Patrik Brundin, David K Simon, Michael Schwarzschild, David Weiner, Jamie Adams, Charles Venuto, Ted M Dawson, Liana Baker, Melissa Kostrzebski, Tina Ward, Gary Rafaloff
Importance: There is a critical need for careful and independent validation of reported symptomatic efficacy and dopaminergic biomarker changes induced by nilotinib in Parkinson disease (PD). Objectives: To assess safety and tolerability of nilotinib in participants with moderately advanced PD. Secondary and exploratory objectives were to assess its affect on PD disability, pharmacokinetics, cerebrospinal fluid (CSF) penetration, and biomarkers. Design, Setting, and Participants: This was a 6-month, multicenter, randomized parallel-group, double-blind, placebo-controlled trial...
March 1, 2021: JAMA Neurology