• Aleksandar Videnovic, Yo-El S Ju, Isabelle Arnulf, Valérie Cochen-De Cock, Birgit Högl, Dieter Kunz, Federica Provini, Pietro-Luca Ratti, Mya C Schiess, Carlos H Schenck, Claudia Trenkwalder, and Treatment and Trials Working Group of the International RBD Study Group.
• Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA AVIDENOVIC@mgh.harvard.edu.
• J. Neurol. Neurosurg. Psychiatr. 2020 Jul 1; 91 (7): 740-749.

AbstractThe rapid eye movement sleep behavioural disorder (RBD) population is an ideal study population for testing disease-modifying treatments for synucleinopathies, since RBD represents an early prodromal stage of synucleinopathy when neuropathology may be more responsive to treatment. While clonazepam and melatonin are most commonly used as symptomatic treatments for RBD, clinical trials of symptomatic treatments are also needed to identify evidence-based treatments. A comprehensive framework for both disease-modifying and symptomatic treatment trials in RBD is described, including potential treatments in the pipeline, cost-effective participant recruitment and selection, study design, outcomes and dissemination of results. For disease-modifying treatment clinical trials, the recommended primary outcome is phenoconversion to an overt synucleinopathy, and stratification features should be used to select a study population at high risk of phenoconversion, to enable more rapid clinical trials. For symptomatic treatment clinical trials, objective polysomnogram-based measurement of RBD-related movements and vocalisations should be the primary outcome measure, rather than subjective scales or diaries. Mobile technology to enable objective measurement of RBD episodes in the ambulatory setting, and advances in imaging, biofluid, tissue, and neurophysiological biomarkers of synucleinopathies, will enable more efficient clinical trials but are still in development. Increasing awareness of RBD among the general public and medical community coupled with timely diagnosis of these diseases will facilitate progress in the development of therapeutics for RBD and associated neurodegenerative disorders.© Author(s) (or their employer(s)) 2020. No commercial re-use. See rights and permissions. Published by BMJ.

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