The study objective is to determine the efficacy gfCoepzyme Q10 (CpQ) in Hjptington's disease (HD). Although the genetic defect that causes HD has been identified there is no known effective treatmentor cure. Rational therapeutic strategies in Huntington's disease include those that are targeted to improving cellular energy production andreducing oxidative stress. Coenzyme Q10, a co-factor involved in mitochondria! electron transfer and an anti-oxidant, is a compound that has some of these properties. Coenzyme Q10 slows progression and prolongs survival in a dose dependent manner in a transgenic mouse model of HD. Coenzyme Q10 at a dosage of 600 mg per day for 2 Vtyears appeared to slowed the functional decline in HD by approximately 13%compared to placebo. Pre-clinical and clinical studies with COQ suggest that higher dosages are more beneficial. The study hypothesis is that chronic treatment of HD patients with CoQ will slow the progressive functional decline of HD.
The specific aim i s to test this hypothesis by conducting a double-blind, placebo-controlled, randomized, parallel group, multi-center study of CoQ involving 608 ambulatory HD subjects who are each treated for 60 months. Eligible subjects will be randomized to CoQ 2400 mg daily or matching placebo. A CoQ dosage of 2400 mg was chosen based on dosage ranging tolerability studies in HD, amyotrophic lateral sclerosis and Parkinson's disease. Theprimary outcome measure will be the clinical progression of HD as measured by the change in the total functional capacity (TFC) between baseline and 60 months. Secondary measures will include changes in the other clinical rating scales of the Unified Huntington Disease Rating Scale (UHDRS), times to decline in TFCby 2 and 3 points, ability to complete the study at the assigned dosage and the frequencies of clinical and laboratory adverse events.

National Institute of Health (NIH)
National Institute of Neurological Disorders and Stroke (NINDS)
Research Project--Cooperative Agreements (U01)
Project #
Application #
Study Section
Special Emphasis Panel (ZNS1-SRB-K (22))
Program Officer
Sutherland, Margaret L
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Massachusetts General Hospital
United States
Zip Code
Alcalay, R N; Wolf, P; Levy, O A et al. (2018) Alpha galactosidase A activity in Parkinson's disease. Neurobiol Dis 112:85-90
Lee, Annie J; Wang, Yuanjia; Alcalay, Roy N et al. (2017) Penetrance estimate of LRRK2 p.G2019S mutation in individuals of non-Ashkenazi Jewish ancestry. Mov Disord 32:1432-1438
Banno, Haruhiko; Andrzejewski, Kelly L; McDermott, Michael P et al. (2017) Analysis of Participant Withdrawal in Huntington Disease Clinical Trials. J Huntingtons Dis 6:149-156
Devanand, Davangere P; Pelton, Gregory H; D'Antonio, Kristina et al. (2017) Low-dose Lithium Treatment for Agitation and Psychosis in Alzheimer Disease and Frontotemporal Dementia: A Case Series. Alzheimer Dis Assoc Disord 31:73-75
Schobel, Scott A; Palermo, Giuseppe; Auinger, Peggy et al. (2017) Motor, cognitive, and functional declines contribute to a single progressive factor in early HD. Neurology 89:2495-2502
Alcalay, Roy N; Levy, Oren A; Wolf, Pavlina et al. (2016) SCARB2 variants and glucocerebrosidase activity in Parkinson's disease. NPJ Parkinsons Dis 2:
Chen, Honglei; Marder, Karen (2016) Milk consumption and the risk of nigral degeneration. Neurology 86:496-7
Terrelonge Jr, Mark; Marder, Karen S; Weintraub, Daniel et al. (2016) CSF ?-Amyloid 1-42 Predicts Progression to Cognitive Impairment in Newly Diagnosed Parkinson Disease. J Mol Neurosci 58:88-92
Mata, Ignacio F; Leverenz, James B; Weintraub, Daniel et al. (2016) GBA Variants are associated with a distinct pattern of cognitive deficits in Parkinson's disease. Mov Disord 31:95-102
Alcalay, Roy N; Mejia-Santana, Helen; Mirelman, Anat et al. (2015) Neuropsychological performance in LRRK2 G2019S carriers with Parkinson's disease. Parkinsonism Relat Disord 21:106-10

Showing the most recent 10 out of 21 publications