Randomized Controlled Trial

. 2017 Jan 10;88(2):152-159.

doi: 10.1212/WNL.0000000000003478. Epub 2016 Dec 2.

A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease

Andrew McGarry¹, Michael McDermott², Karl Kieburtz², Elisabeth A de Blieck², Flint Beal², Karen Marder², Christopher Ross², Ira Shoulson², Peter Gilbert², William M Mallonee², Mark Guttman², Joanne Wojcieszek², Rajeev Kumar², Mark S LeDoux², Mary Jenkins², H Diana Rosas², Martha Nance², Kevin Biglan², Peter Como², Richard M Dubinsky², Kathleen M Shannon², Padraig O'Suilleabhain², Kelvin Chou², Francis Walker², Wayne Martin², Vicki L Wheelock², Elizabeth McCusker², Joseph Jankovic², Carlos Singer², Juan Sanchez-Ramos², Burton Scott², Oksana Suchowersky², Stewart A Factor², Donald S Higgins Jr², Eric Molho², Fredy Revilla², John N Caviness², Joseph H Friedman², Joel S Perlmutter², Andrew Feigin², Karen Anderson², Ramon Rodriguez², Nikolaus R McFarland², Russell L Margolis², Eric S Farbman², Lynn A Raymond², Valerie Suski², Sandra Kostyk², Amy Colcher², Lauren Seeberger², Eric Epping², Sherali Esmail², Nancy Diaz², Wai Lun Alan Fung², Alan Diamond², Samuel Frank², Philip Hanna², Neal Hermanowicz², Leon S Dure², Merit Cudkowicz²; Huntington Study Group 2CARE Investigators and Coordinators

Collaborators, Affiliations

Collaborators

Huntington Study Group 2CARE Investigators and Coordinators:
John Adams, Mandar Jog, Christopher Hyson, Sarah Furtado, Andrew Duker, Melissa Armstrong, Christian Lachner, Hubert Fernandez, Michael S Okun, David Shprecher, Michael Cartwright, Clement Loy, Joohi Jiminez-Shahed, Bradley Robottom, Gregory Suter, Theresa Moore, Jane Forsyth, Andrea Hurt, Joann Belden, Katie Price, Diane Erickson, Breanna Nickels, Misty M Thompson, Linda Cole, Julie Megens, Emilija Makaji, Sara-Lynn Masse, Keith Malarick, Louisa Mook, Susan Maya, Alex Bender, Jessica Meyer, Puja Turakhia, Katherine Harwood, Kathryn Duderstadt, Sarah Lenarz, Judy Hamerlinck, Patricia Edern, Charlyne Hickey, Ashley Owens, Clare Gibbons, Carolyn Gray, Jean A Jaglin, Kimberly Janko, Holly Lawrence, Barbara Estes, Brigid Hayward, Allison Johnson, Amit Gode, Giselle Huet, Beverly Romero-Kersh, Kristine Wernette, Elizabeth Sullivan, Jamie Guyot, Julie Konkle, Christine O'Neill, Pamela King, Amanda Martin, John Bautista, Nicole Mans, Jane Griffith, Erica Surles, Sharon Halton, Alicia Palao, Nathalie Padron, Kolleen Elliott, Lynn Oelke, London Butterfield, Peggy Perry-Trice, Sarah Wyne, Carol Pantella, Lorelei Tainsh, Elaine Sperin, Sharon Evans, Maureen Gartner, Amy Duffy, Pamela Kristof, Lisa Niles, Steven Rainone, Angie Wernle, Ronda Clouse, Michelle Cines, Kelly Dustin, Maura Deeley, Stacy Merritt, Heather Ferreri, Alison McMurray, Alanna Sheinberg, Marsha Hughes-Gay, Kori Ladonna, Erin Chung, Jennifer Lee, Rachel Goldstein, Lindsay Esposito, Nicholas Scoglio, Matthew Grana, Carol Zimmerman, Lucia M Blasucci, William Thayer, Jennifer Hawkins, Julia Koch, Victoria Hunt, Jessica Bargoil, Ingrid Scott, Terry Tempkin, Linda Stewart, Emily Hayes, Donna Galea, Beatriz Belmar, Kylie Richardson, Christine Hunter, Ernesto Jimenez, Monica Quesada, Wendy Levy, Anita Blenke, Lisa Gauger, Joanna Stoner, Mary Lou Klimek, Barbara Sommerfeld, Mary Eglow, Katy Regan, Erin Neefus, Marie Malikowski, Teri Radam, Marci Zomok, Margaret Lannon, Rhonda Agramonte, Johanna Hartlein, Constance Nickerson, Samantha Gibson, Erin Hastings Monari, Camille Swartz, Anne Smith-Bova, Randi Jones, Catherine Wielinski, Shanthi Graham, David Gunn, Jillian McMillan

Affiliations

¹ From Cooper University Healthcare at Rowan University (A.M.), Camden, NJ; University of Rochester (M.M., K.K., E.A.d.B., K.B., P.C.); Weill Cornell Medical Center (F.B.); Columbia Presbyterian Medical Center (K.M.), New York, NY; Johns Hopkins Medical Center (C.R.), Baltimore, MD; Georgetown University (I.S.), Washington, DC; NINDS (P.G.), Bethesda, MD; Hereditary Neurological Disease Center (W.M.M.), Wichita, KS; The Centre for Movement Disorders (M.G.), Toronto, Canada; Indiana University School of Medicine (J.W.), Indianapolis; Colorado Neurological Institute (R.K.), Englewood; University of Tennessee Health Science Center (M.S.L.), Memphis; London Health Sciences Centre (M.J.), Canada; Massachusetts General Hospital (H.D.R.), Boston; Struthers Parkinson's Center (M.N.), Minneapolis, MN; University of Kansas Medical Center (R.M.D.), Kansas City; Rush University Medical Center (K.M.S.), Chicago, IL; University of Texas Southwestern Medical Center (P.O.), Dallas; University of Michigan (K.C.), Ann Arbor; Wake Forest University (F.W.), Winston-Salem, NC; University of Alberta (W.M.), Edmonton, Canada; University of California Davis (V.L.W.), Sacramento; Westmead Hospital (E. McCusker), Westmead, Australia; Baylor College of Medicine (J.J.), Houston, TX; University of Miami Miller School of Medicine (C.S.), FL; University of South Florida (J.S.-R.), Tampa; Duke University (B.S.), Durham, NC; University of Calgary (O.S.), Canada; Emory University School of Medicine (S.A.F.), Atlanta, GA; Albany Medical College (D.S.H., Eric Molho), NY; University of Cincinnati (F.R.), OH; Mayo Clinic Arizona (J.N.C.), Scottsdale; Butler Hospital (J.H.F.), Providence, RI; Washington University (J.S.P.), St. Louis, MO; Feinstein Institute for Medical Research (A.F.), Manhasset, NY; Georgetown University (K.A.), Washington, DC; University of Florida (R.R., N.R.M.), Gainesville; Johns Hopkins University (R.L.M.), Baltimore, MD; University of Nevada School of Medicine (E.S.F.), Reno; University of British Columbia (L.A.R.), Vancouver, Canada; University of Pittsburgh (V.S.), PA; Ohio State University (S.K.), Columbus; The Cooper University Health System (A.C.), Camden, NJ; Idaho Elks Rehabilitation Hospital (L.S.), Boise, ID; University of Iowa (E.E.), Iowa City; North York General Hospital 1 (S.E.), Toronto, Canada; St. Luke's Hospital (N.D.), Allentown, PA; North York General Hospital 2 (W.L.A.F.), University of Toronto, Canada; Washington Regional Medical Center (A.D.), Fayetteville, AR; Beth-Israel Deaconess Medical Center (S.F.), Boston, MA; NJ Neuroscience Institute (P.H.), Edison; University of California Irvine (N.H.); The University of Alabama at Birmingham (L.S.D.); and Massachusetts General Hospital (M.C.), Harvard Medical School, Boston. mcgarry-andrew@cooperhealth.edu.
² From Cooper University Healthcare at Rowan University (A.M.), Camden, NJ; University of Rochester (M.M., K.K., E.A.d.B., K.B., P.C.); Weill Cornell Medical Center (F.B.); Columbia Presbyterian Medical Center (K.M.), New York, NY; Johns Hopkins Medical Center (C.R.), Baltimore, MD; Georgetown University (I.S.), Washington, DC; NINDS (P.G.), Bethesda, MD; Hereditary Neurological Disease Center (W.M.M.), Wichita, KS; The Centre for Movement Disorders (M.G.), Toronto, Canada; Indiana University School of Medicine (J.W.), Indianapolis; Colorado Neurological Institute (R.K.), Englewood; University of Tennessee Health Science Center (M.S.L.), Memphis; London Health Sciences Centre (M.J.), Canada; Massachusetts General Hospital (H.D.R.), Boston; Struthers Parkinson's Center (M.N.), Minneapolis, MN; University of Kansas Medical Center (R.M.D.), Kansas City; Rush University Medical Center (K.M.S.), Chicago, IL; University of Texas Southwestern Medical Center (P.O.), Dallas; University of Michigan (K.C.), Ann Arbor; Wake Forest University (F.W.), Winston-Salem, NC; University of Alberta (W.M.), Edmonton, Canada; University of California Davis (V.L.W.), Sacramento; Westmead Hospital (E. McCusker), Westmead, Australia; Baylor College of Medicine (J.J.), Houston, TX; University of Miami Miller School of Medicine (C.S.), FL; University of South Florida (J.S.-R.), Tampa; Duke University (B.S.), Durham, NC; University of Calgary (O.S.), Canada; Emory University School of Medicine (S.A.F.), Atlanta, GA; Albany Medical College (D.S.H., Eric Molho), NY; University of Cincinnati (F.R.), OH; Mayo Clinic Arizona (J.N.C.), Scottsdale; Butler Hospital (J.H.F.), Providence, RI; Washington University (J.S.P.), St. Louis, MO; Feinstein Institute for Medical Research (A.F.), Manhasset, NY; Georgetown University (K.A.), Washington, DC; University of Florida (R.R., N.R.M.), Gainesville; Johns Hopkins University (R.L.M.), Baltimore, MD; University of Nevada School of Medicine (E.S.F.), Reno; University of British Columbia (L.A.R.), Vancouver, Canada; University of Pittsburgh (V.S.), PA; Ohio State University (S.K.), Columbus; The Cooper University Health System (A.C.), Camden, NJ; Idaho Elks Rehabilitation Hospital (L.S.), Boise, ID; University of Iowa (E.E.), Iowa City; North York General Hospital 1 (S.E.), Toronto, Canada; St. Luke's Hospital (N.D.), Allentown, PA; North York General Hospital 2 (W.L.A.F.), University of Toronto, Canada; Washington Regional Medical Center (A.D.), Fayetteville, AR; Beth-Israel Deaconess Medical Center (S.F.), Boston, MA; NJ Neuroscience Institute (P.H.), Edison; University of California Irvine (N.H.); The University of Alabama at Birmingham (L.S.D.); and Massachusetts General Hospital (M.C.), Harvard Medical School, Boston.

PMID: 27913695
PMCID: PMC5224719
DOI: 10.1212/WNL.0000000000003478

Randomized Controlled Trial

A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease

Andrew McGarry et al. Neurology. 2017.

. 2017 Jan 10;88(2):152-159.

doi: 10.1212/WNL.0000000000003478. Epub 2016 Dec 2.

Authors

Collaborators

Huntington Study Group 2CARE Investigators and Coordinators:
John Adams, Mandar Jog, Christopher Hyson, Sarah Furtado, Andrew Duker, Melissa Armstrong, Christian Lachner, Hubert Fernandez, Michael S Okun, David Shprecher, Michael Cartwright, Clement Loy, Joohi Jiminez-Shahed, Bradley Robottom, Gregory Suter, Theresa Moore, Jane Forsyth, Andrea Hurt, Joann Belden, Katie Price, Diane Erickson, Breanna Nickels, Misty M Thompson, Linda Cole, Julie Megens, Emilija Makaji, Sara-Lynn Masse, Keith Malarick, Louisa Mook, Susan Maya, Alex Bender, Jessica Meyer, Puja Turakhia, Katherine Harwood, Kathryn Duderstadt, Sarah Lenarz, Judy Hamerlinck, Patricia Edern, Charlyne Hickey, Ashley Owens, Clare Gibbons, Carolyn Gray, Jean A Jaglin, Kimberly Janko, Holly Lawrence, Barbara Estes, Brigid Hayward, Allison Johnson, Amit Gode, Giselle Huet, Beverly Romero-Kersh, Kristine Wernette, Elizabeth Sullivan, Jamie Guyot, Julie Konkle, Christine O'Neill, Pamela King, Amanda Martin, John Bautista, Nicole Mans, Jane Griffith, Erica Surles, Sharon Halton, Alicia Palao, Nathalie Padron, Kolleen Elliott, Lynn Oelke, London Butterfield, Peggy Perry-Trice, Sarah Wyne, Carol Pantella, Lorelei Tainsh, Elaine Sperin, Sharon Evans, Maureen Gartner, Amy Duffy, Pamela Kristof, Lisa Niles, Steven Rainone, Angie Wernle, Ronda Clouse, Michelle Cines, Kelly Dustin, Maura Deeley, Stacy Merritt, Heather Ferreri, Alison McMurray, Alanna Sheinberg, Marsha Hughes-Gay, Kori Ladonna, Erin Chung, Jennifer Lee, Rachel Goldstein, Lindsay Esposito, Nicholas Scoglio, Matthew Grana, Carol Zimmerman, Lucia M Blasucci, William Thayer, Jennifer Hawkins, Julia Koch, Victoria Hunt, Jessica Bargoil, Ingrid Scott, Terry Tempkin, Linda Stewart, Emily Hayes, Donna Galea, Beatriz Belmar, Kylie Richardson, Christine Hunter, Ernesto Jimenez, Monica Quesada, Wendy Levy, Anita Blenke, Lisa Gauger, Joanna Stoner, Mary Lou Klimek, Barbara Sommerfeld, Mary Eglow, Katy Regan, Erin Neefus, Marie Malikowski, Teri Radam, Marci Zomok, Margaret Lannon, Rhonda Agramonte, Johanna Hartlein, Constance Nickerson, Samantha Gibson, Erin Hastings Monari, Camille Swartz, Anne Smith-Bova, Randi Jones, Catherine Wielinski, Shanthi Graham, David Gunn, Jillian McMillan

Affiliations

¹ From Cooper University Healthcare at Rowan University (A.M.), Camden, NJ; University of Rochester (M.M., K.K., E.A.d.B., K.B., P.C.); Weill Cornell Medical Center (F.B.); Columbia Presbyterian Medical Center (K.M.), New York, NY; Johns Hopkins Medical Center (C.R.), Baltimore, MD; Georgetown University (I.S.), Washington, DC; NINDS (P.G.), Bethesda, MD; Hereditary Neurological Disease Center (W.M.M.), Wichita, KS; The Centre for Movement Disorders (M.G.), Toronto, Canada; Indiana University School of Medicine (J.W.), Indianapolis; Colorado Neurological Institute (R.K.), Englewood; University of Tennessee Health Science Center (M.S.L.), Memphis; London Health Sciences Centre (M.J.), Canada; Massachusetts General Hospital (H.D.R.), Boston; Struthers Parkinson's Center (M.N.), Minneapolis, MN; University of Kansas Medical Center (R.M.D.), Kansas City; Rush University Medical Center (K.M.S.), Chicago, IL; University of Texas Southwestern Medical Center (P.O.), Dallas; University of Michigan (K.C.), Ann Arbor; Wake Forest University (F.W.), Winston-Salem, NC; University of Alberta (W.M.), Edmonton, Canada; University of California Davis (V.L.W.), Sacramento; Westmead Hospital (E. McCusker), Westmead, Australia; Baylor College of Medicine (J.J.), Houston, TX; University of Miami Miller School of Medicine (C.S.), FL; University of South Florida (J.S.-R.), Tampa; Duke University (B.S.), Durham, NC; University of Calgary (O.S.), Canada; Emory University School of Medicine (S.A.F.), Atlanta, GA; Albany Medical College (D.S.H., Eric Molho), NY; University of Cincinnati (F.R.), OH; Mayo Clinic Arizona (J.N.C.), Scottsdale; Butler Hospital (J.H.F.), Providence, RI; Washington University (J.S.P.), St. Louis, MO; Feinstein Institute for Medical Research (A.F.), Manhasset, NY; Georgetown University (K.A.), Washington, DC; University of Florida (R.R., N.R.M.), Gainesville; Johns Hopkins University (R.L.M.), Baltimore, MD; University of Nevada School of Medicine (E.S.F.), Reno; University of British Columbia (L.A.R.), Vancouver, Canada; University of Pittsburgh (V.S.), PA; Ohio State University (S.K.), Columbus; The Cooper University Health System (A.C.), Camden, NJ; Idaho Elks Rehabilitation Hospital (L.S.), Boise, ID; University of Iowa (E.E.), Iowa City; North York General Hospital 1 (S.E.), Toronto, Canada; St. Luke's Hospital (N.D.), Allentown, PA; North York General Hospital 2 (W.L.A.F.), University of Toronto, Canada; Washington Regional Medical Center (A.D.), Fayetteville, AR; Beth-Israel Deaconess Medical Center (S.F.), Boston, MA; NJ Neuroscience Institute (P.H.), Edison; University of California Irvine (N.H.); The University of Alabama at Birmingham (L.S.D.); and Massachusetts General Hospital (M.C.), Harvard Medical School, Boston. mcgarry-andrew@cooperhealth.edu.
² From Cooper University Healthcare at Rowan University (A.M.), Camden, NJ; University of Rochester (M.M., K.K., E.A.d.B., K.B., P.C.); Weill Cornell Medical Center (F.B.); Columbia Presbyterian Medical Center (K.M.), New York, NY; Johns Hopkins Medical Center (C.R.), Baltimore, MD; Georgetown University (I.S.), Washington, DC; NINDS (P.G.), Bethesda, MD; Hereditary Neurological Disease Center (W.M.M.), Wichita, KS; The Centre for Movement Disorders (M.G.), Toronto, Canada; Indiana University School of Medicine (J.W.), Indianapolis; Colorado Neurological Institute (R.K.), Englewood; University of Tennessee Health Science Center (M.S.L.), Memphis; London Health Sciences Centre (M.J.), Canada; Massachusetts General Hospital (H.D.R.), Boston; Struthers Parkinson's Center (M.N.), Minneapolis, MN; University of Kansas Medical Center (R.M.D.), Kansas City; Rush University Medical Center (K.M.S.), Chicago, IL; University of Texas Southwestern Medical Center (P.O.), Dallas; University of Michigan (K.C.), Ann Arbor; Wake Forest University (F.W.), Winston-Salem, NC; University of Alberta (W.M.), Edmonton, Canada; University of California Davis (V.L.W.), Sacramento; Westmead Hospital (E. McCusker), Westmead, Australia; Baylor College of Medicine (J.J.), Houston, TX; University of Miami Miller School of Medicine (C.S.), FL; University of South Florida (J.S.-R.), Tampa; Duke University (B.S.), Durham, NC; University of Calgary (O.S.), Canada; Emory University School of Medicine (S.A.F.), Atlanta, GA; Albany Medical College (D.S.H., Eric Molho), NY; University of Cincinnati (F.R.), OH; Mayo Clinic Arizona (J.N.C.), Scottsdale; Butler Hospital (J.H.F.), Providence, RI; Washington University (J.S.P.), St. Louis, MO; Feinstein Institute for Medical Research (A.F.), Manhasset, NY; Georgetown University (K.A.), Washington, DC; University of Florida (R.R., N.R.M.), Gainesville; Johns Hopkins University (R.L.M.), Baltimore, MD; University of Nevada School of Medicine (E.S.F.), Reno; University of British Columbia (L.A.R.), Vancouver, Canada; University of Pittsburgh (V.S.), PA; Ohio State University (S.K.), Columbus; The Cooper University Health System (A.C.), Camden, NJ; Idaho Elks Rehabilitation Hospital (L.S.), Boise, ID; University of Iowa (E.E.), Iowa City; North York General Hospital 1 (S.E.), Toronto, Canada; St. Luke's Hospital (N.D.), Allentown, PA; North York General Hospital 2 (W.L.A.F.), University of Toronto, Canada; Washington Regional Medical Center (A.D.), Fayetteville, AR; Beth-Israel Deaconess Medical Center (S.F.), Boston, MA; NJ Neuroscience Institute (P.H.), Edison; University of California Irvine (N.H.); The University of Alabama at Birmingham (L.S.D.); and Massachusetts General Hospital (M.C.), Harvard Medical School, Boston.

PMID: 27913695
PMCID: PMC5224719
DOI: 10.1212/WNL.0000000000003478

Abstract

Objective: To test the hypothesis that chronic treatment of early-stage Huntington disease (HD) with high-dose coenzyme Q10 (CoQ) will slow the progressive functional decline of HD.

Methods: We performed a multicenter randomized, double-blind, placebo-controlled trial. Patients with early-stage HD (n = 609) were enrolled at 48 sites in the United States, Canada, and Australia from 2008 to 2012. Patients were randomized to receive either CoQ 2,400 mg/d or matching placebo, then followed for 60 months. The primary outcome variable was the change from baseline to month 60 in Total Functional Capacity score (for patients who survived) combined with time to death (for patients who died) analyzed using a joint-rank analysis approach.

Results: An interim analysis for futility revealed a conditional power of <5% for the primary analysis, prompting premature conclusion in July 2014. No statistically significant differences were seen between treatment groups for the primary or secondary outcome measures. CoQ was generally safe and well-tolerated throughout the study.

Conclusions: These data do not justify use of CoQ as a treatment to slow functional decline in HD.

Clinicaltrialsgov identifier: NCT00608881.

Classification of evidence: This article provides Class I evidence that CoQ does not slow the progressive functional decline of patients with HD.

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Figures

**Figure 1. Patient disposition**
AE = adverse event; CoQ = coenzyme Q10.

See this image and copyright information in PMC

Comment in

Is mitochondrial oxidative metabolism the right therapy target in early Huntington disease?
Lodi R. Lodi R. Neurology. 2017 Jan 10;88(2):116-117. doi: 10.1212/WNL.0000000000003497. Epub 2016 Dec 2. Neurology. 2017. PMID: 27913697 No abstract available.

References

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1. Rüb U, Vonsettel JP, Heinsen H, Korf HW. The neuropathology of Huntington's disease: classical findings, recent developments and correlation to functional neuroanatomy. Adv Anat Embryol Cell Biol 2015;217:1–146. - PubMed
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A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease

Collaborators

Affiliations

A randomized, double-blind, placebo-controlled trial of coenzyme Q10 in Huntington disease

Authors

Collaborators

Affiliations

Abstract

Figures

Comment in

References

Publication types

MeSH terms

Substances

Associated data

Grants and funding

LinkOut - more resources

Full Text Sources

Other Literature Sources

Medical

Research Materials