%0 Journal Article
%A Höglinger, Günter U
%A Schöpe, Jakob
%A Stamelou, Maria
%A Kassubek, Jan
%A Del Ser, Teodoro
%A Boxer, Adam L
%A Wagenpfeil, Stefan
%A Huppertz, Hans-Jürgen
%A Investigators, AL-108-231
%A Investigators, Tauros MRI
%A Group, Movement Disorder Society-Endorsed PSP Study
%T Longitudinal magnetic resonance imaging in progressive supranuclear palsy: A new combined score for clinical trials.
%J Movement disorders
%V 32
%N 6
%@ 0885-3185
%C New York, NY
%I Wiley
%M DZNE-2020-05636
%P 842-852
%D 2017
%X Two recent, randomized, placebo-controlled phase II/III trials (clinicaltrials.gov: NCT01110720, NCT01049399) of davunetide and tideglusib in progressive supranuclear palsy (PSP) generated prospective, 1-year longitudinal datasets of high-resolution T1-weighted three-dimensional MRI.The objective of this study was to develop a quantitative MRI disease progression measurement for clinical trials.The authors performed a fully automated quantitative MRI analysis employing atlas-based volumetry and provide sample size calculations based on data collected in 99 PSP patients assigned to placebo in these trials. Based on individual volumes of 44 brain compartments and structures at baseline and 52 weeks of follow-up, means and standard deviations of annualized percentage volume changes were used to estimate standardized effect sizes and the required sample sizes per group for future 2-armed, placebo-controlled therapeutic trials.The highest standardized effect sizes were found for midbrain, frontal lobes, and the third ventricle. Using the annualized percentage volume change of these structures to detect a 50
%K Aged
%K Clinical Trials as Topic: methods
%K Disease Progression
%K Female
%K Frontal Lobe: diagnostic imaging
%K Humans
%K Longitudinal Studies
%K Magnetic Resonance Imaging: methods
%K Male
%K Mesencephalon: diagnostic imaging
%K Middle Aged
%K Supranuclear Palsy, Progressive: diagnostic imaging
%K Third Ventricle: diagnostic imaging
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:28436538
%2 pmc:PMC5808453
%R 10.1002/mds.26973
%U https://pub.dzne.de/record/139314