title: Interventional trials in atypical parkinsonism.
abstract: Atypical parkinson disorders (APD) are rapidly progressive neurodegenerative diseases with a variable clinical presentation that may even mimic Parkinson's disease. Multiple system atrophy (MSA), progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) are commonly summarized under this umbrella term. Significant developments in research have expanded knowledge and have broadened available symptomatic treatments, particularly for the treatment of neurogenic orthostatic hypotension. Nonetheless, symptomatic support still remains limited in all of these disorders. Currently, there exists no effective treatment to delay disease progression and disease-modifying trials have failed to provide coherent and convincing results. Recent trials of rasagiline (in MSA), rifampicin (in MSA), tideglusib (in PSP) and davunetide (in PSP) reported negative results. Nevertheless, large cohorts of patients were recruited for interventional studies in the last few years which improved our understanding of trial methodology in APDs immensely. In addition, remarkable progress in basic research has been reported recently and will provide a solid foundation for future therapeutic trials. In this review, we will summarize published randomized, placebo-controlled clinical trials (RCTs) in APDs. Additionally, the design of ongoing and unpublished interventions will be presented.
authors.affiliation: Department of Neurology, Medical University of Innsbruck, Anichstraße 35, 6020, Innsbruck, Austria.
authors.forename: S
authors.initials: S
authors.lastname: Eschlböck
authors.affiliation: Department of Neurology, Medical University of Innsbruck, Anichstraße 35, 6020, Innsbruck, Austria.
authors.forename: F
authors.initials: F
authors.lastname: Krismer
authors.affiliation: Department of Neurology, Medical University of Innsbruck, Anichstraße 35, 6020, Innsbruck, Austria. Electronic address: gregor.wenning@i-med.ac.at.
authors.forename: G K
authors.initials: GK
authors.lastname: Wenning
mesh_terms: D001480:Basal Ganglia Diseases / Q000175:diagnosis / Q000378:metabolism / Q000628:therapy; D002135:Calcium-Binding Proteins / Q000037:antagonists & inhibitors / Q000378:metabolism; D003937:Diagnosis, Differential; D061345:Early Medical Intervention / Q000379:methods*; D006801:Humans; D008565:Membrane Proteins / Q000037:antagonists & inhibitors / Q000378:metabolism; D019578:Multiple System Atrophy / Q000175:diagnosis / Q000378:metabolism / Q000628:therapy; D009124:Muscle Proteins / Q000037:antagonists & inhibitors / Q000378:metabolism; D010300:Parkinson Disease / Q000175:diagnosis* / Q000378:metabolism / Q000628:therapy*; D020734:Parkinsonian Disorders / Q000175:diagnosis / Q000378:metabolism / Q000628:therapy; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D013494:Supranuclear Palsy, Progressive / Q000175:diagnosis / Q000378:metabolism / Q000628:therapy
chemical_list: D002135:Calcium-Binding Proteins; D008565:Membrane Proteins; D009124:Muscle Proteins; C091825:syntrophin alpha1
keywords: Clinical trials; Corticobasal degeneration; Multiple system atrophy; Progressive supranuclear palsy; Therapies