OBJECTIVE: To determine if future studies of coenzyme Q(10) and GPI-1485 in Parkinson disease (PD) may be warranted. METHODS: We conducted a randomized, double-blind, calibrated futility clinical trial of coenzyme Q10 and GPI-1485 in early untreated PD using placebo data from the DATATOP study to establish the futility threshold. RESULTS: The primary outcome measure (change in total Unified Parkinson's Disease Rating Scale scores over 1 year) did not meet the prespecified criteria for futility for either agent. Secondary analyses using calibration controls and other more recent placebo data question the appropriateness of the predetermined definition of futility, and suggest that a more restrictive threshold may be needed. CONCLUSIONS: Coenzyme Q(10) and GPI-1485 may warrant further study in Parkinson disease, although the data are inconsistent. Additional factors (cost, availability of other agents, more recent data on placebo outcomes, other ongoing trials) should also be considered in the selection of agents for Phase III studies.
RCT Entities:
OBJECTIVE: To determine if future studies of coenzyme Q(10) and GPI-1485 in Parkinson disease (PD) may be warranted. METHODS: We conducted a randomized, double-blind, calibrated futility clinical trial of coenzyme Q10 and GPI-1485 in early untreated PD using placebo data from the DATATOP study to establish the futility threshold. RESULTS: The primary outcome measure (change in total Unified Parkinson's Disease Rating Scale scores over 1 year) did not meet the prespecified criteria for futility for either agent. Secondary analyses using calibration controls and other more recent placebo data question the appropriateness of the predetermined definition of futility, and suggest that a more restrictive threshold may be needed. CONCLUSIONS:Coenzyme Q(10) and GPI-1485 may warrant further study in Parkinson disease, although the data are inconsistent. Additional factors (cost, availability of other agents, more recent data on placebo outcomes, other ongoing trials) should also be considered in the selection of agents for Phase III studies.
Authors: Karl Kieburtz; Barbara C Tilley; Jordan J Elm; Debra Babcock; Robert Hauser; G Webster Ross; Alicia H Augustine; Erika U Augustine; Michael J Aminoff; Ivan G Bodis-Wollner; James Boyd; Franca Cambi; Kelvin Chou; Chadwick W Christine; Michelle Cines; Nabila Dahodwala; Lorelei Derwent; Richard B Dewey; Katherine Hawthorne; David J Houghton; Cornelia Kamp; Maureen Leehey; Mark F Lew; Grace S Lin Liang; Sheng T Luo; Zoltan Mari; John C Morgan; Sotirios Parashos; Adriana Pérez; Helen Petrovitch; Suja Rajan; Sue Reichwein; Jessie Tatsuno Roth; Jay S Schneider; Kathleen M Shannon; David K Simon; Tanya Simuni; Carlos Singer; Lewis Sudarsky; Caroline M Tanner; Chizoba C Umeh; Karen Williams; Anne-Marie Wills Journal: JAMA Date: 2015-02-10 Impact factor: 56.272
Authors: David K Simon; Christopher J Swearingen; Robert A Hauser; Joel M Trugman; Michael J Aminoff; Carlos Singer; Daniel Truong; Barbara C Tilley Journal: Clin Neuropharmacol Date: 2008 Jul-Aug Impact factor: 1.592
Authors: Myra G Schneider; Christopher J Swearingen; Lisa M Shulman; Jian Ye; Mona Baumgarten; Barbara C Tilley Journal: Parkinsonism Relat Disord Date: 2008-08-09 Impact factor: 4.891
Authors: Wendy R Galpern; Christopher S Coffey; Alberto Albanese; Ken Cheung; Cynthia L Comella; Dixie J Ecklund; Stanley Fahn; Joseph Jankovic; Karl Kieburtz; Anthony E Lang; Michael P McDermott; Jeremy M Shefner; Jan K Teller; John L P Thompson; Sharon D Yeatts; H A Jinnah Journal: Neurotherapeutics Date: 2014-01 Impact factor: 7.620
Authors: Sydney E Seidel; Barbara C Tilley; Peng Huang; Yuko Y Palesch; Kenneth J Bergmann; Christopher G Goetz; Christopher J Swearingen Journal: Parkinsonism Relat Disord Date: 2011-10-21 Impact factor: 4.891