Coenzyme Q10 in Huntington's Disease (HD) (2CARE)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
University of Rochester
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT00608881
First received: February 4, 2008
Last updated: May 2, 2014
Last verified: May 2014
  Purpose

The goals of this trial are to determine if coenzyme Q10 is effective in slowing the worsening symptoms of Huntington's disease and to learn about the safety and acceptability of long-term coenzyme Q10 use by determining its effects on people with Huntington's disease.


Condition Intervention Phase
Huntington's Disease
Drug: coenzyme Q10
Other: placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: Coenzyme Q10 in Huntington's Disease (HD)

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Change in total functional capacity [ Time Frame: over 5 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in other UHDRS scores; Tolerability - proportion of subjects completing the study at the assigned dosage level; Safety - frequency of adverse events; Times to decline in TFC by 2 and 3 points [ Time Frame: duration of the trial ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 608
Study Start Date: March 2008
Estimated Study Completion Date: September 2017
Estimated Primary Completion Date: August 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: A
Randomized to active treatment (coenzyme Q10 2400 mg/day)
Drug: coenzyme Q10
4 - 300 mg CoQ chewable wafers taken orally twice a day
Other Name: CoQ
Placebo Comparator: B
Randomized to placebo
Other: placebo
an inactive substance

Detailed Description:

Huntington's disease (HD) is a slowly progressive disorder that devastates the lives of those affected and their families. There are no treatments that slow the progression of HD, only mildly effective symptomatic therapies are available.

The purpose of this trial is to find out if coenzyme Q10 (CoQ) is effective in slowing the worsening symptoms of HD. In this study, researchers also will learn about the safety and acceptability of long-term CoQ use by determining its effects on people with HD.

Participants in this trial will be randomly chosen to one of two groups. Group 1 will receive CoQ (2400 mg/day), and group 2 will receive a placebo (an inactive substance). Researchers will compare the change in total functional capacity (TFC)—a measure of functional disability—in the two groups. The TFC is a valid and reliable measure of disease progression and is particularly responsive to change in the early and mid-stages of HD. Researchers will also compare the changes in other components of the Unified Huntington's Disease Rating Scale '99 (UHDRS) including: the total motor score, total behavioral frequency score, total behavior frequency X severity score, verbal fluency test, symbol digit modalities test, Stroop, interference test, functional checklist, and independence scale scores. The groups will also be compared with respect to tolerability, adverse events, vital signs, and laboratory test results as measures of safety.

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

To be eligible for enrollment into this study, subjects must meet the following eligibility criteria within 28 days prior to randomization:

  • Subjects must have clinical features of HD and a confirmed family history of HD, OR a CAG repeat expansion ≥ 36.
  • TFC > 9.
  • Must be ambulatory and not require skilled nursing care.
  • Age ≥ 16 years.
  • Women must not be able to become pregnant (e.g., post menopausal, surgically sterile or using adequate birth control methods for the duration of the study).
  • If psychotropic medications are taken (e.g., anxiolytics, hypnotics, benzodiazepines, antidepressants), they must be at a stable dosage for four weeks prior to randomization and should be maintained at a constant dosage throughout the study, as possible. (Note: stable dosing of tetrabenazine is allowable.) Any changes to these medications mandated by clinical conditions will be systematically recorded and the subject will be permitted to remain in the trial.
  • Able to give informed consent and comply with trial procedures
  • Able to take oral medication.
  • May be required to identify an informant or caregiver who will be willing and able to supervise the daily dosing of study medications and to maintain control of study medications in the home.
  • A designated individual will be identified by the subject to participate in the ongoing consent process should the subject's cognitive capacity to consent become compromised during participation in the study.

Exclusion Criteria:

  • History or known sensitivity of intolerability to CoQ.
  • Exposure to any investigational drug within 30 days of the Baseline visit.
  • Clinical evidence of unstable medical illness in the investigator's judgment.
  • Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression or suicidal ideation within 90 days of the Baseline visit.
  • Substance (alcohol or drug) abuse within one year of the Baseline visit.
  • Women who are pregnant or breastfeeding.
  • Use of supplemental coenzyme Q10 within 30 days prior to the Baseline visit
  • Clinically serious abnormalities in the screening laboratory studies (Screening creatinine greater than 2.0, alanine aminotransferase (ALT) or total bilirubin greater than 3 times the upper limit of normal, absolute neutrophil count of ≤1000/ul, platelet concentration of <100,000/ul, hematocrit level of <33 for female or <35 for male, or coagulation tests > 1.5 time upper limit of normal).
  • Known allergy to FD&C yellow #5 or any other ingredient in the study drug (active and placebo)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00608881

  Hide Study Locations
Locations
United States, Alabama
University of Alabama At Birmingham, Pediatric Neurology Childrens, Harbor Bldg Suite 314, 1600 7Th Avenue South
Birmingham, Alabama, United States, 35233-1711
United States, Arizona
Mayo Clinic Arizona, 13400 East Shea Boulevard, Csu-Cp21B
Scottsdale, Arizona, United States, 85259
United States, Arkansas
WASHINGTON REGIONAL MEDICAL CENTER, 3215 N. North Hills Blvd
Fayetteville, Arkansas, United States, 72703
United States, California
University of California Irvine, Department of Neurology, 100 Irvine Hall
Irvine, California, United States, 92697-4275
University of California Davis, Medical Center Dept of Neurology, Acc Building Suite 3700, 4860 Y Street
Sacramento, California, United States, 95817
United States, Colorado
Colorado Neurological Institute, Movement Disorders Center, 701 East Hampden Avenue Suite 510
Littleton, Colorado, United States, 80120
United States, Florida
University of Florida Center for Movement Disorders and Neurorestoration, 3450 Hull Road, 4th Floor
Gainesville, Florida, United States, 32607
UNIVERSITY OF MIAMI, 1150 NW 14th STREET, #401
Miami, Florida, United States, 33136
University of South Florida, College of Medicine Dept of Neurology, 12901 Bruce B Downs Blvd Mdc-55
Tampa, Florida, United States, 33612
United States, Georgia
Emory University, Wesley Woods Center, 1841 Clifton Road NE Room 314
Atlanta, Georgia, United States, 30329
United States, Idaho
Idaho Elks Rehabilitation Hospital, 600 North Robbins Road
Boise, Idaho, United States, 83702
United States, Illinois
Rush University Medical Center, Department of Neurological Sciences, 1725 West Harrison Suite 755
Chicago, Illinois, United States, 60612
United States, Indiana
Indiana University School of Medicine, Outpatient Clinical Research Facility, 535 Barnhill Drive Room #150
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa Hospital and Clinics, 200 Hawkins Road, Room W263 General Hospital
Iowa City, Iowa, United States, 52242-1000
United States, Kansas
University of Kansas Medical Center, Department of Neurology, 3599 Rainbow Blvd Mail Stop 2012
Kansas City, Kansas, United States, 66160-7314
Hereditary Neurological Disease Centre (Hndc),3223 N. Webb, Suite 4
Wichita, Kansas, United States, 67226
United States, Maryland
Johns Hopkins University, 600 North Wolfe Street, Meyer 2-181
Baltimore, Maryland, United States, 21287
University of Maryland School of Medicine, 22 South Greene Street, N4 W49-B
Baltimore, Maryland, United States, 21201
United States, Massachusetts
Boston University School of Medicine, Department of Neurology, 715 Albany Street C329
Boston, Massachusetts, United States, 02118
Massachusetts General Hospital, 149 13Th Street Suite 2241
Charlestown, Massachusetts, United States, 02129
United States, Michigan
University of Michigan, 1500 E Medical Center Drive, B1 H202 Nuclear Medicine
Ann Arbor, Michigan, United States, 48109-0028
United States, Minnesota
Struthers Parkinson'S Center, 6701 Country Club Drive
Golden Valley, Minnesota, United States, 55427
United States, Missouri
Washington University School of Medicine, Box 8111, 660 South Euclid
St Louis, Missouri, United States, 63110
United States, Nevada
University of Las Vegas School of Medicine, 1707 W. Charleston Blvd, Suite 220
Las Vegas, Nevada, United States, 89102
United States, New Jersey
Cooper University Hospital
Camden, New Jersey, United States, 08103
Nj Neuroscience Institute, Jfk Medical Center, 65 James Street
Edison, New Jersey, United States, 08818
United States, New York
Albany Medical College, Parkinson'S Disease & Movement Disorders Ctr
Albany, New York, United States, 12208
North Shore-Lij Health System, 350 Community Drive Room 110, Research Institute
Manhasset, New York, United States, 11030
Columbia University, Sergievsky Center P&S Box 16, 630 West 168Th Street
New York, New York, United States, 10032
University of Rochester, Department of Neurology, 919 Westfall Road Building C Suite 220
Rochester, New York, United States, 14618
United States, North Carolina
Duke University, 932 Morreene Road #213
Durham, North Carolina, United States, 27705
Wake Forest University, Baptist Med Center, Department of Neurology, Medical Center Boulevard
Winston Salem, North Carolina, United States, 27157
United States, Ohio
University of Cincinnati/Cincinnati Children'S Hospital, 222 Piedmont Avenue, Suite 3200
Cincinnati, Ohio, United States, 45219
OHIO STATE UNIVERSITY , 2006 Kenny Road
Columbus, Ohio, United States, 43212
United States, Pennsylvania
ST. LUKE'S HOSPITAL, 240 Centronia Road
Allentown, Pennsylvania, United States, 18104
University of Pennsylvania, Pennsylvania Hospital Department of Neurology , 330 South 9Th Street
Philadelphia, Pennsylvania, United States, 19107
University of Pittsburgh Kaufmann Medical Building, 3471 Fifth Avunue, Suite 811
Pittsburgh, Pennsylvania, United States, 15213
United States, Rhode Island
BUTLER HOSPTIAL MOVEMENT DISORDER PROGRAM, 345 Blackstone Boulevard
Providence, Rhode Island, United States, 02906
United States, Tennessee
The University of Tennesee Health Science Cen, 855 Monroe Avenue, Department of Neurology, Room 415 Link Bldg
Memphis, Tennessee, United States, 38163
United States, Texas
UN oF TEXAS SOUTHWESTERN MED CENTER DALLAS, 5323 HARRY HINES BOULEVARD H1.108
Dallas, Texas, United States, 75390-9016
Baylor College of Medicine, 6550 Fannin Suite 1801
Houston, Texas, United States, 77030
Australia, New South Wales
Westmead Hospital, Department of Neurology Level 1, Po Box 533
Wentworthville, New South Wales, Australia, 2145
Canada, Alberta
University of Calgary, Heritage Medical Research Clinic, Trw Bldg 5 Floor, 3280 Hospital Dri. NW
Calgary, Alberta, Canada, T2N 4Z6
University of Alberta, Glenrose Rehab Hosp, Movement Disorder Clinic , Rm 0601 Gleneast 10230 - 111 Avenue
Edmonton, Alberta, Canada, T5G 0B7
Canada, British Columbia
Department of Medical Genetics, Ubc Hospital, Room S179-2211 Westbrook Mall
Vancouver, British Columbia, Canada, V6T 2B5
Canada, Ontario
London Health Sciences Centre, University Hospital, 339 Windermere Road
London, Ontario, Canada, N6A 5A5
Centre For Movement Disorders, 2780 Bur Oak Avenue
Markham, Ontario, Canada, L4A 1G8
North York General Hospital, 4001 Leslie Street
Toronto, Ontario, Canada, M2R 1N5
NORTH YORK GENERAL HOSPITAL (2), 4001 Leslie Street
Toronto, Ontario, Canada, M2K 1E1
Sponsors and Collaborators
Massachusetts General Hospital
University of Rochester
Investigators
Principal Investigator: Merit Cudkowicz, MD MSc Massachusetts General Hospital
Principal Investigator: Michael McDermott, PhD University of Rochester, Biostatistics
Principal Investigator: Karl Kieburtz, MD MPH Director, Clinical Trials Coordination Center, University of Rochester
  More Information

Publications:
Kowall N, Ferrante R, Martin J. Patterns of cell loss in Huntington's disease. Trends in Neurosciences 1987;10:24-29.
Riley D, Lang A. Movement Disorders. In: Bradley W, Daroff R, Fenichel G, eds. Neurology in Clinical Practice. The Neurological Disorders. Boston: Butterworth-Heinemann, 1991: 1563-1601.
Harper P. The epidemiology of Huntington's disease. Human Genetics 1992;89:365-376.
Bruyn G. Huntington's chorea: Historical clinical and laboratory synopsis. In: Vinken P, Bruyn G, eds. Handbook of Clinical Neurology. Amsterdam, 1968: 298-378.
Greenamyre J, Shoulson I. Huntington's Disease. In: Calne D, ed. Neurodegenerative Diseases. Philadelphia: WB Saunders, 1994: 685-704.
Feigin A, Kieburtz K, Bordwell K, et al. Functional decline in Huntington's disease. Movement Disorders 1995;10:211-214.
Kido D, Shoulson I, Manzione J, Harnish P. Measurement of caudate nucleus and putamen atrophy in patients with Huntington's disease. Neuroradiology 1991;33:604-606.
Pepping J. Co-enyzme Q10. American Journal of Health-System Pharmacy 1999;56:519-521.
Yamagami T, Okishio T, Toyama S, Kishi T. Correlation of serum coenzyme Q10 level and leukocute complex II activity in nformal and cardiovascular patients. In: Folkers K, Yamagami T, eds. Biomedical and clinical aspects of coenzyme Q: Elsevier Science Publishers, 1981: 79-89.
Dubois B, Brand M, Garcia de Yebenes J, et al. European-Huntington's-disease-Initiative (EHDI)-Trial: Objectives, design, and description of the study population at the end of inclusion. Mov Dis 2002;17:S319.
Bogentoft C, Edelund P, Olsson B, Widlund L, Westensen K. Biopharmaceutical aspects of intraveneous and oral administration of coenzyme Q10. In: Folkers K, Littarru G, Yamagami T, eds. Biomedical and clinical aspects of coenzyme Q.: Elsevier Science Publishers, 1991: 215-224.
Lucker P, Wetselsberg N, Hennings G, Rehn D. Pharmacokinetics of coenzyme ubidecarenone in healthy volunteers. In: Folkers K, Littarru G, Yamagami T, eds. Biomedical and clinical aspects of coenzyme Q: Elsevier Science Publishers, 1984: 143-151.
Weber C. Dietary intake and absorption of coenzyme Q. In: Kagan V, Quinn P, eds. Coenzyme Q: Molecular Mechanisms in Health and Disease. Boca Raton: CRC Press, 2001:209-215.
Saito Y, Kubo H, Bujo H, Yamamoto Y. The changes in plasma coenzyme Q10 level during the statin therapy for hypercholesterolemic patients. In: Second Conference of the International Coenzyme Q10 Association.; 2000, 2000: 59.

Responsible Party: Merit E. Cudkowicz, MD, Julieanne Dorn Professor of Neurology, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT00608881     History of Changes
Other Study ID Numbers: 2CARE 01.00, 5U01NS052592, 5R01NS052619
Study First Received: February 4, 2008
Last Updated: May 2, 2014
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada

Keywords provided by Massachusetts General Hospital:
Huntington's disease
Huntington disease
HD
coenzyme Q10
CoQ

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Chorea
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Mental Disorders
Coenzyme Q10
Ubiquinone
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Vitamins

ClinicalTrials.gov processed this record on September 11, 2014