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

被引：4

作者：

McGarry, Andrew ^{[1
]}

McDermott, Michael ^{[2
]}

Kieburtz, Karl ^{[2
]}

de Blieck, Elisabeth A. ^{[2
]}

Beal, Flint ^{[3
]}

Marder, Karen ^{[4
]}

Ross, Christopher ^{[5
]}

Shoulson, Ira ^{[6
]}

Gilbert, Peter ^{[7
]}

Mallonee, William M. ^{[8
]}

Guttman, Mark ^{[9
]}

Wojcieszek, Joanne ^{[10
]}

Kumar, Rajeev ^{[11
]}

LeDoux, Mark S. ^{[12
]}

Jenkins, Mary ^{[13
]}

Rosas, H. Diana ^{[14
]}

Nance, Martha ^{[15
]}

Biglan, Kevin ^{[2
]}

Como, Peter ^{[2
]}

Dubinsky, Richard M. ^{[16
]}

Shannon, Kathleen M. ^{[17
]}

O'Suilleabhain, Padraig ^{[18
]}

Chou, Kelvin ^{[19
]}

Walker, Francis ^{[20
]}

Martin, Wayne ^{[21
]}

Wheelock, Vicki L. ^{[22
]}

McCusker, Elizabeth ^{[23
]}

Jankovic, Joseph ^{[24
]}

Singer, Carlos ^{[25
]}

Sanchez-Ramos, Juan ^{[26
]}

Scott, Burton ^{[27
]}

Suchowersky, Oksana ^{[28
]}

Factor, Stewart A. ^{[29
]}

Higgins, Donald S., Jr. ^{[30
]}

Molho, Eric ^{[30
]}

Revilla, Fredy ^{[31
]}

Caviness, John N. ^{[32
]}

Friedman, Joseph H. ^{[33
]}

Perlmutter, Joel S. ^{[34
]}

Feigin, Andrew ^{[35
]}

Anderson, Karen ^{[6
]}

Rodriguez, Ramon ^{[36
]}

McFarland, Nikolaus R. ^{[36
]}

Margolis, Russell L. ^{[37
]}

Farbman, Eric S. ^{[38
]}

Raymond, Lynn A. ^{[39
]}

Suski, Valerie ^{[40
]}

Kostyk, Sandra ^{[41
]}

Colcher, Amy ^{[42
]}

Seeberger, Lauren ^{[43
]}

机构：

[1] Rowan Univ, Cooper Univ Healthcare, Camden, NJ 08102 USA

[2] Univ Rochester, Rochester, NY 14627 USA

[3] Weill Cornell Med Ctr, New York, NY USA

[4] Columbia Presbyterian Med Ctr, New York, NY 10032 USA

[5] Johns Hopkins Med Ctr, Baltimore, MD USA

[6] Georgetown Univ, Washington, DC USA

[7] NINDS, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

[8] Hereditary Neurol Dis Ctr, Wichita, KS USA

[9] Ctr Movement Disorders, Toronto, ON, Canada

[10] Indiana Univ Sch Med, Indianapolis, IN 46202 USA

[11] Colorado Neurol Inst, Englewood, CO USA

[12] Univ Tennessee, Ctr Hlth Sci, Memphis, TN 38163 USA

[13] London Hlth Sci Ctr, London, ON, Canada

[14] Massachusetts Gen Hosp, Boston, MA 02114 USA

[15] Struthers Parkinsons Ctr, Minneapolis, MN USA

[16] Univ Kansas Med Ctr, Kansas City, KS USA

[17] Rush Univ Med Ctr, Chicago, IL USA

[18] Univ Texas Southwestern Med Ctr, Dallas, TX USA

[19] Univ Michigan, Ann Arbor, MI 48109 USA

[20] Wake Forest Univ, Winston Salem, NC 27109 USA

[21] Univ Alberta, Edmonton, AB, Canada

[22] Univ Calif Davis, Sacramento, CA 95817 USA

[23] Westmead Hosp, Westmead, NSW, Australia

[24] Baylor Coll Med, Houston, TX 77030 USA

[25] Univ Miami, Miller Sch Med, Coral Gables, FL 33124 USA

[26] Univ S Florida, Tampa, FL USA

[27] Duke Univ, Durham, NC USA

[28] Univ Calgary, Calgary, AB T2N 1N4, Canada

[29] Emory Univ, Sch Med, Atlanta, GA USA

[30] Albany Med Coll, Albany, NY 12208 USA

[31] Univ Cincinnati, Cincinnati, OH 45221 USA

[32] Mayo Clin Arizona, Scottsdale, AZ USA

[33] Butler Hosp, Providence, RI 02906 USA

[34] Washington Univ, St Louis, MO USA

[35] Feinstein Inst Med Res, Manhasset, NY USA

[36] Univ Florida, Gainesville, FL USA

[37] Johns Hopkins Univ, Baltimore, MD USA

[38] Univ Nevada, Sch Med, Reno, NV 89557 USA

[39] Univ British Columbia, Vancouver, BC, Canada

[40] Univ Pittsburgh, Pittsburgh, PA 15260 USA

[41] Ohio State Univ, Columbus, OH 43210 USA

[42] Cooper Univ Hlth Syst, Camden, NJ USA

[43] Idaho Elks Rehabil Hosp, Boise, ID USA

[44] Univ Iowa, Iowa City, IA USA

[45] North York Gen Hosp 1, Toronto, ON, Canada

[46] St Lukes Hosp, Allentown, PA USA

[47] Univ Toronto, North York Gen Hosp 2, Toronto, ON M5S 1A1, Canada

[48] Washington Reg Med Ctr, Fayetteville, AR USA

[49] Beth Israel Deaconess Med Ctr, Boston, MA 02215 USA

[50] NJ Neurosci Inst, Edison, NJ USA

来源：

NEUROLOGY | 2017年 / 88卷 / 02期

关键词：

ENERGY-METABOLISM; CLINICAL-TRIALS; IN-VIVO; Q(10); CREATINE; IMPAIRMENT; REMACEMIDE; DEFECTS;

D O I：

10.1212/wnl.0000000000003478

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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. ClinicalTrials.gov identifier: NCT00608881. Classification of evidence: This article provides Class I evidence that CoQ does not slow the progressive functional decline of patients with HD. Neurology (R) 2017;88:152-159

引用

页码：152 / 159

页数：8

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