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000276156 037__ $$aDZNE-2025-00228
000276156 041__ $$aEnglish
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000276156 1001_ $$aGermani, Massimiliano$$b0
000276156 245__ $$aCombined Assessment of Function and Survival to Demonstrate the Effect of Treatment on Progressive Supranuclear Palsy.
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000276156 500__ $$aThis research was funded by UCB. G.H. was supported by the European Joint Programme on Rare Diseases (Improve-PSP) and the Deutsche Forschungsgemeinschaft DFG, (German Research Foundation) under Germany's Excellence Strategy within the framework of the Munich Cluster for Systems Neurology (EXC 2145 SyNergy ID 390857198).
000276156 520__ $$aProgressive supranuclear palsy (PSP) is a rare and fatal neurodegenerative disorder for which there are currently no disease-modifying treatments. Recent trials of potential therapies had durations of 12 months, which may be insufficient because of nonrandom missingness due to death. Longer durations, incorporating PSP Rating Scale and survival, can reduce the potential for type II error. Selecting efficacy measures more sensitive to disease modification may facilitate identification of treatment effect.The objective of this study was to evaluate the simulated phase 3 PSP trial assessing the effect of disease-modifying intervention on a novel combined primary endpoint comprising function (PSP Rating Scale) and survival, the Combined Assessment of Function and Survival (CAFS), and to determine operating characteristics of the CAFS.To simulate PSP progression in the trial population, we developed models of PSP Rating Scale and survival using data from published clinical studies. These models were used to define operating characteristics of the CAFS for use in a phase 3 trial.The sample size determined (N = 384; 1:1 randomization) would provide >80% power to detect significant treatment effects on the CAFS compared with placebo. The CAFS provides good operating characteristics and increased power to detect moderate treatment effects on the PSP Rating Scale. We propose a trial design allowing potential detection of treatment effects at a preplanned interim analysis after participants complete 12 months of treatment, with assessment of effects of treatment (≤24 months) on survival.Use of the CAFS could provide a comprehensive and robust estimate of the clinical benefit of future therapies. © 2024 UCB. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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000276156 650_7 $$2Other$$aclinical rating scale
000276156 650_7 $$2Other$$aitem response theory
000276156 650_7 $$2Other$$ajoint rank test
000276156 650_7 $$2Other$$aneurodegenerative disease
000276156 650_7 $$2Other$$aprogressive supranuclear palsy
000276156 650_2 $$2MeSH$$aSupranuclear Palsy, Progressive: drug therapy
000276156 650_2 $$2MeSH$$aSupranuclear Palsy, Progressive: physiopathology
000276156 650_2 $$2MeSH$$aHumans
000276156 650_2 $$2MeSH$$aDisease Progression
000276156 650_2 $$2MeSH$$aMale
000276156 650_2 $$2MeSH$$aFemale
000276156 650_2 $$2MeSH$$aTreatment Outcome
000276156 650_2 $$2MeSH$$aSeverity of Illness Index
000276156 650_2 $$2MeSH$$aAged
000276156 7001_ $$00009-0004-0635-2485$$aRebollo Mesa, Irene$$b1
000276156 7001_ $$aBuchanan, Tim J$$b2
000276156 7001_ $$aDe Bruyn, Steven$$b3
000276156 7001_ $$aGasalla, Teresa$$b4
000276156 7001_ $$aVan Tricht, Hans Lieve G$$b5
000276156 7001_ $$aEwen, Colin$$b6
000276156 7001_ $$aGolbe, Lawrence I$$b7
000276156 7001_ $$aBoxer, Adam$$b8
000276156 7001_ $$0P:(DE-2719)2811373$$aHöglinger, Günter$$b9$$eLast author$$udzne
000276156 773__ $$0PERI:(DE-600)2041249-6$$a10.1002/mds.30027$$gVol. 40, no. 1, p. 97 - 107$$n1$$p97 - 107$$tMovement disorders$$v40$$x0885-3185$$y2025
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