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

February 29, 2016 updated by: Merit E. Cudkowicz, MD, Massachusetts General Hospital
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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

609

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: A - coenzyme Q10 2400 mg/day
Randomized to active treatment (coenzyme Q10 2400 mg/day)
Drug: coenzyme Q10
4 - 300 mg CoQ chewable wafers taken orally twice a day
Other Names:
  • CoQ
Placebo Comparator: B - Placebo
Randomized to placebo
Other: placebo
an inactive substance

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Joint Rank (Combination of Time to Death (for Subjects Who Died) and Change in Total Functional Capacity Score (TFC) From Baseline to Month 60 (for Subjects Who Survived))
Time Frame: 5 years
The primary outcome variable at the start of the trial was the change in TFC score from baseline to Month 60. The Data and Safety Monitoring Board recommended to the trial leadership that they reconsider how they accommodate missing data from subjects who die in their primary analysis of the change in TFC score. Based on these recommendations, the trial leadership changed the primary analysis to that of a joint rank approach. TFC consists of five ordinally scaled items assessing a person's capacity with: (1) occupation; (2) financial affairs; (3) domestic responsibilities; (4) activities of daily living; and (5) independent living. Total score ranges from zero (worst) to 13 (best).
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Total Functional Capacity (TFC) Score From Baseline to Month 60
Time Frame: Baseline and Month 60
TFC consists of five ordinally scaled items assessing a person's capacity with: (1) occupation; (2) financial affairs; (3) domestic responsibilities; (4) activities of daily living; and (5) independent living. Total score ranges from zero (worst) to 13 (best).
Baseline and Month 60
Change in Functional Checklist Score From Baseline to Month 60
Time Frame: Baseline and Month 60
The functional assessment checklist includes 25 questions about common daily tasks. A score of 1 is given for each "yes" reply and a score of 0 is given for each "no" reply (scale range is 0-25). Higher scores indicate better functioning.
Baseline and Month 60
Change in Independence Scale Score From Baseline to Month 60
Time Frame: Baseline and Month 60
The independence scale assesses independence on a 0 to 100 scale with higher scores indicating better functioning.
Baseline and Month 60
Change in Total Motor Score From Baseline to Month 60
Time Frame: Baseline and Month 60
The motor section of the Unified Huntington's Disease Rating Scale (UHDRS) assesses motor features of Huntington disease with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. The total motor score is the sum of all the individual motor ratings, with higher scores (124) indicating more severe motor impairment than lower scores. The score ranges from 0 to 124.
Baseline and Month 60
Change in Behavioral Frequency Score From Baseline to Month 60
Time Frame: Baseline and Month 60
The Unified Huntington's Disease Rating Scale (UHDRS) behavioral subscale assesses frequency and severity of psychiatric-related symptoms, including depressed mood, apathy, low self-esteem/guilt, suicidal thoughts, anxiety, irritable behavior, aggressive behavior, obsessional thinking, compulsive behavior, delusions, and hallucinations. A total score was calculated by summing up all the individual behavioral frequency items (range 0-56) with higher scores representing more severe behavioral impairment.
Baseline and Month 60
Change in Behavioral Frequency x Severity Score From Baseline to Month 60
Time Frame: Baseline and Month 60
The Unified Huntington's Disease Rating Scale (UHDRS) behavioral subscale assesses frequency and severity of psychiatric-related symptoms, including depressed mood, apathy, low self-esteem/guilt, suicidal thoughts, anxiety, irritable behavior, aggressive behavior, obsessional thinking, compulsive behavior, delusions, and hallucinations. The total score is the sum of the product of the individual behavioral frequency and severity items (range 0-176) with higher scores representing more severe behavioral impairment.
Baseline and Month 60
Change in Symbol Digit Modalities Test (SDMT) From Baseline to Month 60
Time Frame: Baseline and Month 60
The SDMT assesses attention, visuoperceptual processing, working memory, and cognitive/psychomotor speed. The score is the number of correctly paired abstract symbols and specific numbers in 90 seconds with higher scores indicating better cognitive functioning.
Baseline and Month 60
Change in Verbal Fluency Test From Baseline to Month 60
Time Frame: Baseline and Month 60
The verbal fluency test is typically considered a measure of executive function. The score is the number of correct words produced across three 1-minute trials.
Baseline and Month 60
Change in Stroop Interference Test - Color Naming From Baseline to Month 60
Time Frame: Baseline and Month 60
Stroop Interference Test - color naming score is the total number of correct colors identified in 45 seconds and reflects processing speed.
Baseline and Month 60
Change in Stroop Interference Test - Word Reading From Baseline to Month 60
Time Frame: Baseline and Month 60
Stroop Interference Test - word reading score is the total number of correct words read in 45 seconds and reflects processing speed.
Baseline and Month 60
Change in Stroop Interference Test - Interference From Baseline to Month 60
Time Frame: Baseline and Month 60
Stroop Interference Test - interference score is the total number of correct items identified in 45 seconds and reflects an executive measure of inhibitory ability.
Baseline and Month 60
Time to a Two-Point Decline in TFC Score or Death
Time Frame: 5 years
TFC consists of five ordinally scaled items assessing a person's capacity with: (1) occupation; (2) financial affairs; (3) domestic responsibilities; (4) activities of daily living; and (5) independent living. Total score ranges from zero (worst) to 13 (best).
5 years
Time to a Three-Point Decline in TFC Score or Death
Time Frame: 5 years
TFC consists of five ordinally scaled items assessing a person's capacity with: (1) occupation; (2) financial affairs; (3) domestic responsibilities; (4) activities of daily living; and (5) independent living. Total score ranges from zero (worst) to 13 (best).
5 years
Number Completing Study at Assigned Dosage Level
Time Frame: 5 years
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts General Hospital

Collaborators

National Institute of Neurological Disorders and Stroke (NINDS)
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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2008

Primary Completion (Actual)

November 1, 2014

Study Completion (Actual)

May 1, 2015

Study Registration Dates

First Submitted

February 4, 2008

First Submitted That Met QC Criteria

February 4, 2008

First Posted (Estimate)

February 6, 2008

Study Record Updates

Last Update Posted (Estimate)

March 30, 2016

Last Update Submitted That Met QC Criteria

February 29, 2016

Last Verified

February 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2CARE 01.00
  • 5U01NS052592 (U.S. NIH Grant/Contract)
  • 5R01NS052619 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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