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

167 Citations (Scopus)

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

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coenzyme Q10
Medical Futility
Parkinson Disease
Randomized Controlled Trials
Placebos
Calibration
Outcome Assessment (Health Care)
Clinical Trials
Costs and Cost Analysis
GPI 1485

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Kieburtz, K., Ravina, B., Galpern, W. R., Tilley, B., Shannon, K., Tanner, C., ... 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

A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease. / 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; Rao, Jayaraman; Cook, Maureen; Aminoff, Michael J.; Christine, Chad; Roth, Jessie; Leehey, Maureen; Bainbridge, Jacci; Ross, G. Webster; Terashita, Stephanie; Singer, Carlos; Perez, Marian A.; Blenke, Anita; Racette, Brad; Deppen, Patricia; Elble, Rodger; Young, Charlene; Stewart, Tracy; Sethi, Kapil; Dill, Buff; Taylor, John; Roberge, Peggy; Dewey, Richard B.; Hayward, Brigid; Jankovic, Joseph; Hunter, Christine; Wooten, Frederick; Keller, Margaret F.; Jennings, Danna; Kelsey, Tammie; Martin, Wayne; McInnes, Germaine; Wojcieszek, Joanne; Belden, Joann; Albin, Roger; Wernette, Kristine; Savitt, Joseph; Dunlop, Becky; Pahwa, Rajesh; Lyons, Kelly E.; Parsons, Amy; Fang, John; Shearon, Dorothy; Feigin, Andrew; Cox, Margaret Marie; Adler, Charles; Lind, Marlene; Scott, Burton; Field, Joanne; Nance, Martha; Peterson, Susan; Burns, Richard S.; Marlor, Lynn; Van Bodis-Wollner, I.; Hayes, Elizabeth; Schneider, Jay; Sendek, Stephanie; Gollomp, Stephen; Vernon, Gwyn; LeWitt, Peter; DeAngelis, Maryan; Simon, David; Paul, Linda; Gorell, Jay; Krstevska, Shana; Flewellen, Marilyn; McCarthy, Sharon; Uitti, Ryan; Turk, Margaret; Bower, James; Torgrimson, Susan; Sabbagh, Marwan; Obradov, Zoran; Juncos, Jorge; Weeks, Mary Louise Musante; Fernandez, Hubert; Brown, Gordon; Elm, Jordan; Guimaraes, Paulo; Huang, Peng; Palesch, Yuko; Kamp, Cornelia; Shinaman, Aileen; Fraser, Debbie; Brocht, Alicia; Bennett, Susan; Weaver, Chris; Baker, Debbie; Olsen, Beverly; Goetz, Christopher; Miyasaki, Janis; Fagan, Susan; Mauldin, Patrick.

In: Neurology, Vol. 68, No. 1, 01.2007, p. 20-28.

Research output: Contribution to journalArticle

Kieburtz, K, Ravina, B, Galpern, WR, Tilley, B, Shannon, K, Tanner, C, Wooten, GF, Hamill, R, Sage, JI, Kosa, E, Watts, RL, Stover, NR, McMurray, R, Lew, MF, Kawai, C, Coffey, D, LeBlanc, P, Carter, J, Brodsky, M, Andrews, P, Siderowf, A, Reichwein, S, Shulman, L, Weiner, WJ, Pabst, K, Jaglin, J, Hauser, R, McClain, T, Delgado, H, Suchowersky, O, Derwent, L, Rao, J, Cook, M, Aminoff, MJ, Christine, C, Roth, J, Leehey, M, Bainbridge, J, Ross, GW, Terashita, S, Singer, C, Perez, MA, Blenke, A, Racette, B, Deppen, P, Elble, R, Young, C, Stewart, T, Sethi, K, Dill, B, Taylor, J, Roberge, P, Dewey, RB, Hayward, B, Jankovic, J, Hunter, C, Wooten, F, Keller, MF, Jennings, D, Kelsey, T, Martin, W, McInnes, G, Wojcieszek, J, Belden, J, Albin, R, Wernette, K, Savitt, J, Dunlop, B, Pahwa, R, Lyons, KE, Parsons, A, Fang, J, Shearon, D, Feigin, A, Cox, MM, Adler, C, Lind, M, Scott, B, Field, J, Nance, M, Peterson, S, Burns, RS, Marlor, L, Van Bodis-Wollner, I, Hayes, E, Schneider, J, Sendek, S, Gollomp, S, Vernon, G, LeWitt, P, DeAngelis, M, Simon, D, Paul, L, Gorell, J, Krstevska, S, Flewellen, M, McCarthy, S, Uitti, R, Turk, M, Bower, J, Torgrimson, S, Sabbagh, M, Obradov, Z, Juncos, J, Weeks, MLM, Fernandez, H, Brown, G, Elm, J, Guimaraes, P, Huang, P, Palesch, Y, Kamp, C, Shinaman, A, Fraser, D, Brocht, A, Bennett, S, Weaver, C, Baker, D, Olsen, B, Goetz, C, Miyasaki, J, Fagan, S & Mauldin, P 2007, 'A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease', Neurology, vol. 68, no. 1, pp. 20-28. https://doi.org/10.1212/01.wnl.0000250355.28474.8e
Kieburtz K, Ravina B, Galpern WR, Tilley B, Shannon K, Tanner C et al. A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease. Neurology. 2007 Jan;68(1):20-28. https://doi.org/10.1212/01.wnl.0000250355.28474.8e
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 ; Rao, Jayaraman ; Cook, Maureen ; Aminoff, Michael J. ; Christine, Chad ; Roth, Jessie ; Leehey, Maureen ; Bainbridge, Jacci ; Ross, G. Webster ; Terashita, Stephanie ; Singer, Carlos ; Perez, Marian A. ; Blenke, Anita ; Racette, Brad ; Deppen, Patricia ; Elble, Rodger ; Young, Charlene ; Stewart, Tracy ; Sethi, Kapil ; Dill, Buff ; Taylor, John ; Roberge, Peggy ; Dewey, Richard B. ; Hayward, Brigid ; Jankovic, Joseph ; Hunter, Christine ; Wooten, Frederick ; Keller, Margaret F. ; Jennings, Danna ; Kelsey, Tammie ; Martin, Wayne ; McInnes, Germaine ; Wojcieszek, Joanne ; Belden, Joann ; Albin, Roger ; Wernette, Kristine ; Savitt, Joseph ; Dunlop, Becky ; Pahwa, Rajesh ; Lyons, Kelly E. ; Parsons, Amy ; Fang, John ; Shearon, Dorothy ; Feigin, Andrew ; Cox, Margaret Marie ; Adler, Charles ; Lind, Marlene ; Scott, Burton ; Field, Joanne ; Nance, Martha ; Peterson, Susan ; Burns, Richard S. ; Marlor, Lynn ; Van Bodis-Wollner, I. ; Hayes, Elizabeth ; Schneider, Jay ; Sendek, Stephanie ; Gollomp, Stephen ; Vernon, Gwyn ; LeWitt, Peter ; DeAngelis, Maryan ; Simon, David ; Paul, Linda ; Gorell, Jay ; Krstevska, Shana ; Flewellen, Marilyn ; McCarthy, Sharon ; Uitti, Ryan ; Turk, Margaret ; Bower, James ; Torgrimson, Susan ; Sabbagh, Marwan ; Obradov, Zoran ; Juncos, Jorge ; Weeks, Mary Louise Musante ; Fernandez, Hubert ; Brown, Gordon ; Elm, Jordan ; Guimaraes, Paulo ; Huang, Peng ; Palesch, Yuko ; Kamp, Cornelia ; Shinaman, Aileen ; Fraser, Debbie ; Brocht, Alicia ; Bennett, Susan ; Weaver, Chris ; Baker, Debbie ; Olsen, Beverly ; Goetz, Christopher ; Miyasaki, Janis ; Fagan, Susan ; Mauldin, Patrick. / A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease. In: Neurology. 2007 ; Vol. 68, No. 1. pp. 20-28.
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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.",
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T1 - A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease

AU - Kieburtz, Karl

AU - Ravina, Bernard

AU - Galpern, Wendy R.

AU - Tilley, Barbara

AU - Shannon, Kathleen

AU - Tanner, Caroline

AU - Wooten, G. Frederick

AU - Hamill, Robert

AU - Sage, Jacob I.

AU - Kosa, Emily

AU - Watts, Ray L.

AU - Stover, Natividad R.

AU - McMurray, Rebecca

AU - Lew, Mark F.

AU - Kawai, Connie

AU - Coffey, David

AU - LeBlanc, Pauline

AU - Carter, Julie

AU - Brodsky, Matthew

AU - Andrews, Pamela

AU - Siderowf, Andrew

AU - Reichwein, Sue

AU - Shulman, Lisa

AU - Weiner, William J.

AU - Pabst, Katharine

AU - Jaglin, Jeana

AU - Hauser, Robert

AU - McClain, Theresa

AU - Delgado, Holly

AU - Suchowersky, Oksana

AU - Derwent, Lorelei

AU - Rao, Jayaraman

AU - Cook, Maureen

AU - Aminoff, Michael J.

AU - Christine, Chad

AU - Roth, Jessie

AU - Leehey, Maureen

AU - Bainbridge, Jacci

AU - Ross, G. Webster

AU - Terashita, Stephanie

AU - Singer, Carlos

AU - Perez, Marian A.

AU - Blenke, Anita

AU - Racette, Brad

AU - Deppen, Patricia

AU - Elble, Rodger

AU - Young, Charlene

AU - Stewart, Tracy

AU - Sethi, Kapil

AU - Dill, Buff

AU - Taylor, John

AU - Roberge, Peggy

AU - Dewey, Richard B.

AU - Hayward, Brigid

AU - Jankovic, Joseph

AU - Hunter, Christine

AU - Wooten, Frederick

AU - Keller, Margaret F.

AU - Jennings, Danna

AU - Kelsey, Tammie

AU - Martin, Wayne

AU - McInnes, Germaine

AU - Wojcieszek, Joanne

AU - Belden, Joann

AU - Albin, Roger

AU - Wernette, Kristine

AU - Savitt, Joseph

AU - Dunlop, Becky

AU - Pahwa, Rajesh

AU - Lyons, Kelly E.

AU - Parsons, Amy

AU - Fang, John

AU - Shearon, Dorothy

AU - Feigin, Andrew

AU - Cox, Margaret Marie

AU - Adler, Charles

AU - Lind, Marlene

AU - Scott, Burton

AU - Field, Joanne

AU - Nance, Martha

AU - Peterson, Susan

AU - Burns, Richard S.

AU - Marlor, Lynn

AU - Van Bodis-Wollner, I.

AU - Hayes, Elizabeth

AU - Schneider, Jay

AU - Sendek, Stephanie

AU - Gollomp, Stephen

AU - Vernon, Gwyn

AU - LeWitt, Peter

AU - DeAngelis, Maryan

AU - Simon, David

AU - Paul, Linda

AU - Gorell, Jay

AU - Krstevska, Shana

AU - Flewellen, Marilyn

AU - McCarthy, Sharon

AU - Uitti, Ryan

AU - Turk, Margaret

AU - Bower, James

AU - Torgrimson, Susan

AU - Sabbagh, Marwan

AU - Obradov, Zoran

AU - Juncos, Jorge

AU - Weeks, Mary Louise Musante

AU - Fernandez, Hubert

AU - Brown, Gordon

AU - Elm, Jordan

AU - Guimaraes, Paulo

AU - Huang, Peng

AU - Palesch, Yuko

AU - Kamp, Cornelia

AU - Shinaman, Aileen

AU - Fraser, Debbie

AU - Brocht, Alicia

AU - Bennett, Susan

AU - Weaver, Chris

AU - Baker, Debbie

AU - Olsen, Beverly

AU - Goetz, Christopher

AU - Miyasaki, Janis

AU - Fagan, Susan

AU - Mauldin, Patrick

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N2 - 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.

AB - 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.

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