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Document type:
journal article 
Author(s):
Stamelou, Maria; Schöpe, Jakob; Wagenpfeil, Stefan; Del Ser, Teodoro; Bang, Jee; Lobach, Iryna Y; Luong, Phi; Respondek, Gesine; Oertel, Wolfgang H; Boxer, AdamL; Höglinger, Günter U; AL-108-231 Investigators, Tauros Investigators, and MDS-Endorsed PSP Study Group 
Title:
Power calculations and placebo effect for future clinical trials in progressive supranuclear palsy. 
Abstract:
Two recent randomized, placebo-controlled trials of putative disease-modifying agents (davunetide, tideglusib) in progressive supranuclear palsy (PSP) failed to show efficacy, but generated data relevant for future trials.We provide sample size calculations based on data collected in 187 PSP patients assigned to placebo in these trials. A placebo effect was calculated.The total PSP-Rating Scale required the least number of patients per group (N = 51) to detect a 50% change in the 1-year progression and 39 when including patients with<= 5 years disease duration. The Schwab and England Activities of Daily Living required 70 patients per group and was highly correlated with the PSP-Rating Scale. A placebo effect was not detected in these scales.We propose the 1-year PSP-Rating Scale score change as the single primary readout in clinical neuroprotective or disease-modifying trials. The Schwab and England Activities of Daily Living could be used as a secondary outcome. © 2016 International Parkinson and Movement Disorder Society. 
Journal title abbreviation:
Mov Disord 
Year:
2016 
Journal volume:
31 
Journal issue:
Pages contribution:
742-7 
Language:
eng 
Fulltext / DOI:
Print-ISSN:
0885-3185 
TUM Institution:
Neurologische Klinik und Poliklinik