Title

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

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