A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease

Authors

• Karl Kieburtz, University of Rochester
• Bernard Ravina, National Institutes of Health (NIH)
• Wendy R. Galpern, National Institutes of Health (NIH)
• Barbara Tilley, Medical University of South Carolina
• Kathleen Shannon, Rush University
• Caroline Tanner, Parkinson's Institute
• G. Frederick Wooten, University of Virginia
• Robert Hamill, University of Vermont
• Jacob I. Sage, Rutgers Robert Wood Johnson Medical School at New Brunswick
• Emily Kosa, Rutgers Robert Wood Johnson Medical School at New Brunswick
• Ray L. Watts, The University of Alabama at Birmingham
• Natividad R. Stover, The University of Alabama at Birmingham
• Rebecca McMurray, The University of Alabama at Birmingham
• Mark F. Lew, University of Southern California
• Connie Kawai, University of Southern California
• David Coffey, Dartmouth-Hitchcock Medical Center
• Pauline LeBlanc, Dartmouth-Hitchcock Medical Center
• Julie Carter, Oregon Health & Science University
• Matthew Brodsky, Oregon Health & Science University
• Pamela Andrews, Oregon Health & Science University
• Andrew Siderowf, University of Pennsylvania
• Sue Reichwein, University of Pennsylvania
• Lisa Shulman, University of Maryland, Baltimore (UMB)
• William J. Weiner, University of Maryland, Baltimore (UMB)
• Katharine Pabst, University of Maryland, Baltimore (UMB)
• Jeana Jaglin, Rush University
• Robert Hauser, University of South Florida, Tampa
• Theresa McClain, University of South Florida, Tampa
• Holly Delgado, University of South Florida, Tampa
• Oksana Suchowersky, University of Calgary
• Lorelei Derwent, University of Calgary
• Jayaraman Rao, Louisiana State University

Document Type

Article

Abstract

OBJECTIVE: To determine if future studies of coenzyme Q10 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 Q10 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. ©2007 AAN Enterprises, Inc.

Publication Date

1-1-2007

Publication Title

Neurology

ISSN

00283878

Volume

68

Issue

1

First Page

20

Last Page

28

PubMed ID

17200487

Digital Object Identifier (DOI)

10.1212/01.wnl.0000250355.28474.8e

Recommended Citation

Kieburtz, Karl; Ravina, Bernard; Galpern, Wendy R.; Tilley, Barbara; Shannon, Kathleen; Tanner, Caroline; Wooten, G. Frederick; Hamill, Robert; Sage, Jacob I.; Kosa, Emily; Watts, Ray L.; Stover, Natividad R.; McMurray, Rebecca; Lew, Mark F.; Kawai, Connie; Coffey, David; LeBlanc, Pauline; Carter, Julie; Brodsky, Matthew; Andrews, Pamela; Siderowf, Andrew; Reichwein, Sue; Shulman, Lisa; Weiner, William J.; Pabst, Katharine; Jaglin, Jeana; Hauser, Robert; McClain, Theresa; Delgado, Holly; Suchowersky, Oksana; Derwent, Lorelei; and Rao, Jayaraman, "A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease" (2007). Neurology. 730.
https://scholar.barrowneuro.org/neurology/730