title: A responsive outcome for Parkinson's disease neuroprotection futility studies.
abstract: Futility studies are designed to test new treatments over a short period in a small number of subjects to determine if those treatments are worthy of larger and longer term studies, or if they should be abandoned. An appropriate outcome measure for a neuroprotection futility study in Parkinson's disease (sensitive to tracking disease progression in the short-term) has not been determined. Data sets from three clinical trials were used to compare Parkinson's disease outcome measures. Total Unified Parkinson's Disease Rating Scale (UPDRS; Mentation + Activities of Daily Living + Motor) change and Motor plus Activities of Daily Living UPDRS change, measured in untreated patients, required the smallest sample sizes of all the outcome measures explored. Other outcomes (UPDRS Motor, UPDRS Activities of Daily Living, and time to need levodopa) required somewhat larger sample sizes. Futility designs in Parkinson's disease are feasible in terms of short duration and small sample size requirements, and this design is being applied in two ongoing Parkinson's disease studies to select agents for future larger and longer term neuroprotection studies.
authors.affiliation: Department of Biostatistics, Bioinformatics and Epidemiology, Medical University of South Carolina, Charleston, SC 29425, USA. elmj@musc.edu
authors.forename: Jordan J
authors.initials: JJ
authors.lastname: Elm
authors.forename: Christopher G
authors.initials: CG
authors.lastname: Goetz
authors.forename: Bernard
authors.initials: B
authors.lastname: Ravina
authors.forename: Kathleen
authors.initials: K
authors.lastname: Shannon
authors.forename: George Fredrick
authors.initials: GF
authors.lastname: Wooten
authors.forename: Caroline M
authors.initials: CM
authors.lastname: Tanner
authors.forename: Yuko Y
authors.initials: YY
authors.lastname: Palesch
authors.forename: Peng
authors.initials: P
authors.lastname: Huang
authors.forename: Paulo
authors.initials: P
authors.lastname: Guimaraes
authors.forename: Cornelia
authors.initials: C
authors.lastname: Kamp
authors.forename: Barbara C
authors.initials: BC
authors.lastname: Tilley
authors.forename: Karl
authors.initials: K
authors.lastname: Kieburtz
mesh_terms: D002986:Clinical Trials as Topic; D018450:Disease Progression; D015259:Dopamine Agents / Q000627:therapeutic use*; D006801:Humans; D018696:Neuroprotective Agents / Q000627:therapeutic use*; D017063:Outcome Assessment, Health Care / Q000379:methods*; D010300:Parkinson Disease / Q000188:drug therapy*
chemical_list: D015259:Dopamine Agents; D018696:Neuroprotective Agents