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

Karl Kieburtz, Bernard Ravina, Wendy R. Galpern, Barbara Tilley, Kathleen Shannon, Caroline Tanner, G. Frederick Wooten, Robert Hamill, Jacob I. Sage, Emily Kosa, Ray L. Watts, Natividad R. Stover, Rebecca McMurray, Mark F. Lew, Connie Kawai, David Coffey, Pauline LeBlanc, Julie Carter, Matthew Brodsky, Pamela AndrewsAndrew Siderowf, Sue Reichwein, Lisa Shulman, William J. Weiner, Katharine Pabst, Jeana Jaglin, Robert Hauser, Theresa McClain, Holly Delgado, Oksana Suchowersky, Lorelei Derwent, Jayaraman Rao, Maureen Cook, Michael J. Aminoff, Chad Christine, Jessie Roth, Maureen Leehey, Jacci Bainbridge, G. Webster Ross, Stephanie Terashita, Carlos Singer, Marian A. Perez, Anita Blenke, Brad Racette, Patricia Deppen, Rodger Elble, Charlene Young, Tracy Stewart, Kapil Sethi, Buff Dill, John Taylor, Peggy Roberge, Richard B. Dewey, Brigid Hayward, Joseph Jankovic, Christine Hunter, Frederick Wooten, Margaret F. Keller, Danna Jennings, Tammie Kelsey, Wayne Martin, Germaine McInnes, Joanne Wojcieszek, Joann Belden, Roger Albin, Kristine Wernette, Joseph Savitt, Becky Dunlop, Rajesh Pahwa, Kelly E. Lyons, Amy Parsons, John Fang, Dorothy Shearon, Andrew Feigin, Margaret Marie Cox, Charles Adler, Marlene Lind, Burton Scott, Joanne Field, Martha Nance, Susan Peterson, Richard S. Burns, Lynn Marlor, I. Van Bodis-Wollner, Elizabeth Hayes, Jay Schneider, Stephanie Sendek, Stephen Gollomp, Gwyn Vernon, Peter LeWitt, Maryan DeAngelis, David Simon, Linda Paul, Jay Gorell, Shana Krstevska, Marilyn Flewellen, Sharon McCarthy, Ryan Uitti, Margaret Turk, James Bower, Susan Torgrimson, Marwan Sabbagh, Zoran Obradov, Jorge Juncos, Mary Louise Musante Weeks, Hubert Fernandez, Gordon Brown, Jordan Elm, Paulo Guimaraes, Peng Huang, Yuko Palesch, Cornelia Kamp, Aileen Shinaman, Debbie Fraser, Alicia Brocht, Susan Bennett, Chris Weaver, Debbie Baker, Beverly Olsen, Christopher Goetz, Janis Miyasaki, Susan Fagan, Patrick Mauldin

Research output: Contribution to journalArticle

168 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)20-28
Number of pages9
JournalNeurology
Volume68
Issue number1
DOIs
StatePublished - Jan 2007

ASJC Scopus subject areas

  • Clinical Neurology

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    Kieburtz, K., Ravina, B., Galpern, W. R., Tilley, B., Shannon, K., Tanner, C., Wooten, G. F., Hamill, R., Sage, J. I., Kosa, E., Watts, R. L., Stover, N. R., McMurray, R., Lew, M. F., Kawai, C., Coffey, D., LeBlanc, P., Carter, J., Brodsky, M., ... Mauldin, P. (2007). A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease. Neurology, 68(1), 20-28. https://doi.org/10.1212/01.wnl.0000250355.28474.8e