Huntington's DiseasePhase 32CARENCT00608881
Coenzyme Q10 in Huntington's Disease (HD)
A Phase 3 study testing coenzyme Q10 in people with Huntington's Disease, run by Massachusetts General Hospital at 49 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Huntington's Disease
- What is being tested?
- coenzyme Q10
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 609 participants
- Where
- 49 sitesin 3 countries
- Study dates
- Mar 2008to May 2015
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
Show all 11Show fewer
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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)
Trial locations and contacts
49 sites in 3 countries.
49 sites in 3 countries
United States41 sites
Albany Medical College, Parkinson'S Disease & Movement Disorders Ctr
Albany, New York, 12208
ST. LUKE'S HOSPITAL, 240 Centronia Road
Allentown, Pennsylvania, 18104
University of Michigan, 1500 E Medical Center Drive, B1 H202 Nuclear Medicine
Ann Arbor, Michigan, 48109-0028
Emory University, Wesley Woods Center, 1841 Clifton Road NE Room 314
Atlanta, Georgia, 30329
University of Maryland School of Medicine, 22 South Greene Street, N4 W49-B
Baltimore, Maryland, 21201
Johns Hopkins University, 600 North Wolfe Street, Meyer 2-181
Baltimore, Maryland, 21287
University of Alabama At Birmingham, Pediatric Neurology Childrens, Harbor Bldg Suite 314, 1600 7Th Avenue South
Birmingham, Alabama, 35233-1711
Idaho Elks Rehabilitation Hospital, 600 North Robbins Road
Boise, Idaho, 83702
Boston University School of Medicine, Department of Neurology, 715 Albany Street C329
Boston, Massachusetts, 02118
Cooper University Hospital
Camden, New Jersey, 08103
Massachusetts General Hospital, 149 13Th Street Suite 2241
Charlestown, Massachusetts, 02129
Rush University Medical Center, Department of Neurological Sciences, 1725 West Harrison Suite 755
Chicago, Illinois, 60612
University of Cincinnati/Cincinnati Children'S Hospital, 222 Piedmont Avenue, Suite 3200
Cincinnati, Ohio, 45219
OHIO STATE UNIVERSITY , 2006 Kenny Road
Columbus, Ohio, 43212
UN oF TEXAS SOUTHWESTERN MED CENTER DALLAS, 5323 HARRY HINES BOULEVARD H1.108
Dallas, Texas, 75390-9016
Duke University, 932 Morreene Road #213
Durham, North Carolina, 27705
Nj Neuroscience Institute, Jfk Medical Center, 65 James Street
Edison, New Jersey, 08818
WASHINGTON REGIONAL MEDICAL CENTER, 3215 N. North Hills Blvd
Fayetteville, Arkansas, 72703
University of Florida Center for Movement Disorders and Neurorestoration, 3450 Hull Road, 4th Floor
Gainesville, Florida, 32607
Struthers Parkinson'S Center, 6701 Country Club Drive
Golden Valley, Minnesota, 55427
Baylor College of Medicine, 6550 Fannin Suite 1801
Houston, Texas, 77030
Indiana University School of Medicine, Outpatient Clinical Research Facility, 535 Barnhill Drive Room #150
Indianapolis, Indiana, 46202
University of Iowa Hospital and Clinics, 200 Hawkins Road, Room W263 General Hospital
Iowa City, Iowa, 52242-1000
University of California Irvine, Department of Neurology, 100 Irvine Hall
Irvine, California, 92697-4275
University of Kansas Medical Center, Department of Neurology, 3599 Rainbow Blvd Mail Stop 2012
Kansas City, Kansas, 66160-7314
University of Las Vegas School of Medicine, 1707 W. Charleston Blvd, Suite 220
Las Vegas, Nevada, 89102
Colorado Neurological Institute, Movement Disorders Center, 701 East Hampden Avenue Suite 510
Littleton, Colorado, 80120
North Shore-Lij Health System, 350 Community Drive Room 110, Research Institute
Manhasset, New York, 11030
The University of Tennesee Health Science Cen, 855 Monroe Avenue, Department of Neurology, Room 415 Link Bldg
Memphis, Tennessee, 38163
UNIVERSITY OF MIAMI, 1150 NW 14th STREET, #401
Miami, Florida, 33136
Columbia University, Sergievsky Center P&S Box 16, 630 West 168Th Street
New York, New York, 10032
University of Pennsylvania, Pennsylvania Hospital Department of Neurology , 330 South 9Th Street
Philadelphia, Pennsylvania, 19107
University of Pittsburgh Kaufmann Medical Building, 3471 Fifth Avunue, Suite 811
Pittsburgh, Pennsylvania, 15213
BUTLER HOSPTIAL MOVEMENT DISORDER PROGRAM, 345 Blackstone Boulevard
Providence, Rhode Island, 02906
University of Rochester, Department of Neurology, 919 Westfall Road Building C Suite 220
Rochester, New York, 14618
University of California Davis, Medical Center Dept of Neurology, Acc Building Suite 3700, 4860 Y Street
Sacramento, California, 95817
Mayo Clinic Arizona, 13400 East Shea Boulevard, Csu-Cp21B
Scottsdale, Arizona, 85259
Washington University School of Medicine, Box 8111, 660 South Euclid
St Louis, Missouri, 63110
University of South Florida, College of Medicine Dept of Neurology, 12901 Bruce B Downs Blvd Mdc-55
Tampa, Florida, 33612
Hereditary Neurological Disease Centre (Hndc),3223 N. Webb, Suite 4
Wichita, Kansas, 67226
Wake Forest University, Baptist Med Center, Department of Neurology, Medical Center Boulevard
Winston-Salem, North Carolina, 27157
Canada7 sites
University of Calgary, Heritage Medical Research Clinic, Trw Bldg 5 Floor, 3280 Hospital Dri. NW
Calgary, Alberta, T2N 4Z6
University of Alberta, Glenrose Rehab Hosp, Movement Disorder Clinic , Rm 0601 Gleneast 10230 - 111 Avenue
Edmonton, Alberta, T5G 0B7
London Health Sciences Centre, University Hospital, 339 Windermere Road
London, Ontario, N6A 5A5
Centre For Movement Disorders, 2780 Bur Oak Avenue
Markham, Ontario, L4A 1G8
NORTH YORK GENERAL HOSPITAL (2), 4001 Leslie Street
Toronto, Ontario, M2K 1E1
North York General Hospital, 4001 Leslie Street
Toronto, Ontario, M2R 1N5
Department of Medical Genetics, Ubc Hospital, Room S179-2211 Westbrook Mall
Vancouver, British Columbia, V6T 2B5
Australia1 site
Westmead Hospital, Department of Neurology Level 1, Po Box 533
Wentworthville, New South Wales, 2145
About this study
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.
Read the 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.
- Conditions
- Huntington's Disease
- Being tested
- coenzyme Q10, placebo
- Keywords
- Huntington's disease, Huntington disease, HD, coenzyme Q10, CoQ
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
coenzyme Q10Drug
Also called CoQ
4 - 300 mg CoQ chewable wafers taken orally twice a day
placeboOther
an inactive substance
| Study group | Role | What they receive |
|---|---|---|
| A - coenzyme Q10 2400 mg/day | Active comparator | Randomized to active treatment (coenzyme Q10 2400 mg/day) Receives: Drug: coenzyme Q10 |
| B - Placebo | Placebo comparator | Randomized to placebo Receives: Other: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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))
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).
Measured: 5 years
Secondary outcomes (14)
Change in Total Functional Capacity (TFC) Score From Baseline to 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).
Measured: Baseline and Month 60
Change in Functional Checklist Score From Baseline to 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.
Measured: Baseline and Month 60
Change in Independence Scale Score From Baseline to Month 60
The independence scale assesses independence on a 0 to 100 scale with higher scores indicating better functioning.
Measured: Baseline and Month 60
Change in Total Motor Score From Baseline to 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.
Measured: Baseline and Month 60
Change in Behavioral Frequency Score From Baseline to 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.
Measured: Baseline and Month 60
Change in Behavioral Frequency x Severity Score From Baseline to 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.
Measured: Baseline and Month 60
Change in Symbol Digit Modalities Test (SDMT) From Baseline to 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.
Measured: Baseline and Month 60
Change in Verbal Fluency Test From Baseline to 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.
Measured: Baseline and Month 60
Change in Stroop Interference Test - Color Naming From Baseline to Month 60
Stroop Interference Test - color naming score is the total number of correct colors identified in 45 seconds and reflects processing speed.
Measured: Baseline and Month 60
Change in Stroop Interference Test - Word Reading From Baseline to Month 60
Stroop Interference Test - word reading score is the total number of correct words read in 45 seconds and reflects processing speed.
Measured: Baseline and Month 60
Change in Stroop Interference Test - Interference From Baseline to 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.
Measured: Baseline and Month 60
Time to a Two-Point Decline in TFC Score or Death
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).
Measured: 5 years
Time to a Three-Point Decline in TFC Score or Death
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).
Measured: 5 years
Number Completing Study at Assigned Dosage Level
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2008 (estimated)
- Study startMar 2008
- Primary completionNov 2014
- Study completionMay 2015
Why it stopped: Futility analysis failed to showed likelihoo of benefit of CoQ 2400 mg/day.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (76)Papers the sponsor has linked to this study.
- Other trials for Huntington's DiseaseStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 609 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester
- Responsible party
- Principal investigator, Merit E. Cudkowicz, MD, Massachusetts General Hospital
- Investigators
- Merit Cudkowicz, MD MSc, Principal investigator, Massachusetts General HospitalMichael McDermott, PhD, Principal investigator, University of Rochester, BiostatisticsKarl Kieburtz, MD MPH, Principal investigator, Director, Clinical Trials Coordination Center, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00608881
- Sponsor's protocol ID
- 2CARE 01.00
- Other IDs
- 5U01NS052592, 5R01NS052619
- Acronym
- 2CARE
- Status last verified
- Feb 2016
- First submitted
- 4 Feb 2008
- Last updated
- 30 Mar 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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.
Adams P, Falek A, Arnold J. Huntington disease in Georgia: age at onset. Am J Hum Genet. 1988 Nov;43(5):695-704.
PubMed 2973230Conneally PM. Huntington disease: genetics and epidemiology. Am J Hum Genet. 1984 May;36(3):506-26.
PubMed 6233902Harper PS. The epidemiology of Huntington's disease. Hum Genet. 1992 Jun;89(4):365-76. doi: 10.1007/BF00194305.
PubMed 1535611Tanner CM, Goldman SM. Epidemiology of movement disorders. Curr Opin Neurol. 1994 Aug;7(4):340-5. doi: 10.1097/00019052-199408000-00011. No abstract available.
PubMed 7952243Young AB, Shoulson I, Penney JB, Starosta-Rubinstein S, Gomez F, Travers H, Ramos-Arroyo MA, Snodgrass SR, Bonilla E, Moreno H, et al. Huntington's disease in Venezuela: neurologic features and functional decline. Neurology. 1986 Feb;36(2):244-9. doi: 10.1212/wnl.36.2.244.
PubMed 2935747Bruyn G. Huntington's chorea: Historical clinical and laboratory synopsis. In: Vinken P, Bruyn G, eds. Handbook of Clinical Neurology. Amsterdam, 1968: 298-378.
Leigh RJ, Newman SA, Folstein SE, Lasker AG, Jensen BA. Abnormal ocular motor control in Huntington's disease. Neurology. 1983 Oct;33(10):1268-75. doi: 10.1212/wnl.33.10.1268.
PubMed 6225033Caine ED, Hunt RD, Weingartner H, Ebert MH. Huntington's dementia. Clinical and neuropsychological features. Arch Gen Psychiatry. 1978 Mar;35(3):377-84. doi: 10.1001/archpsyc.1978.01770270127013.
PubMed 153122Bamford KA, Caine ED, Kido DK, Plassche WM, Shoulson I. Clinical-pathologic correlation in Huntington's disease: a neuropsychological and computed tomography study. Neurology. 1989 Jun;39(6):796-801. doi: 10.1212/wnl.39.6.796.
PubMed 2524678Sorensen SA, Fenger K. Causes of death in patients with Huntington's disease and in unaffected first degree relatives. J Med Genet. 1992 Dec;29(12):911-4. doi: 10.1136/jmg.29.12.911.
PubMed 1479606Oliver JE. Huntington's chorea in Northamptonshire. Br J Psychiatry. 1970 Mar;116(532):241-53. doi: 10.1192/bjp.116.532.241. No abstract available.
PubMed 4244787Greenamyre J, Shoulson I. Huntington's Disease. In: Calne D, ed. Neurodegenerative Diseases. Philadelphia: WB Saunders, 1994: 685-704.
Shoulson I, Fahn S. Huntington disease: clinical care and evaluation. Neurology. 1979 Jan;29(1):1-3. doi: 10.1212/wnl.29.1.1. No abstract available.
PubMed 154626
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Coenzyme Q10 in Huntington's Disease (HD)
- ClinicalTrials.gov ID
- NCT00608881
- Acronym / sponsor ID
- 2CARE / 2CARE 01.00
- Conditions
- Huntington's Disease
- Keywords
- Huntington's disease, Huntington disease, HD, coenzyme Q10, CoQ
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Futility analysis failed to showed likelihoo of benefit of CoQ 2400 mg/day.
- Enrolment
- 609 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester
- Study start
- Mar 2008
- Primary completion
- Nov 2014 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 6 Feb 2008 (estimated)
- Results
- Posted 30 Mar 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A - coenzyme Q10 2400 mg/day | Active comparator | Drug: coenzyme Q10Randomized to active treatment (coenzyme Q10 2400 mg/day) |
| B - Placebo | Placebo comparator | Other: placeboRandomized to placebo |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| coenzyme Q10Also: CoQ4 - 300 mg CoQ chewable wafers taken orally twice a day | Drug | A - coenzyme Q10 2400 mg/day |
| placeboan inactive substance | Other | B - Placebo |
Primary outcome
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).
14 secondary outcomes
Change in Total Functional Capacity (TFC) Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Functional Checklist Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Independence Scale Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Total Motor Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Behavioral Frequency Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Behavioral Frequency x Severity Score From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Symbol Digit Modalities Test (SDMT) From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Verbal Fluency Test From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Stroop Interference Test - Color Naming From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Stroop Interference Test - Word Reading From Baseline to Month 60
Time frame: Baseline and Month 60
Change in Stroop Interference Test - Interference From Baseline to Month 60
Time frame: Baseline and Month 60
Time to a Two-Point Decline in TFC Score or Death
Time frame: 5 years
Time to a Three-Point Decline in TFC Score or Death
Time frame: 5 years
Number Completing Study at Assigned Dosage Level
Time frame: 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- BackgroundKowall N, Ferrante R, Martin J. Patterns of cell loss in Huntington's disease. Trends in Neurosciences 1987;10:24-29.
- BackgroundRiley 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.
- Background: PubMed 2973230Adams P, Falek A, Arnold J. Huntington disease in Georgia: age at onset. Am J Hum Genet. 1988 Nov;43(5):695-704.
- Background: PubMed 6233902Conneally PM. Huntington disease: genetics and epidemiology. Am J Hum Genet. 1984 May;36(3):506-26.
- Background: PubMed 1535611Harper PS. The epidemiology of Huntington's disease. Hum Genet. 1992 Jun;89(4):365-76. doi: 10.1007/BF00194305.
- Background: PubMed 7952243Tanner CM, Goldman SM. Epidemiology of movement disorders. Curr Opin Neurol. 1994 Aug;7(4):340-5. doi: 10.1097/00019052-199408000-00011. No abstract available.
- Background: PubMed 2935747Young AB, Shoulson I, Penney JB, Starosta-Rubinstein S, Gomez F, Travers H, Ramos-Arroyo MA, Snodgrass SR, Bonilla E, Moreno H, et al. Huntington's disease in Venezuela: neurologic features and functional decline. Neurology. 1986 Feb;36(2):244-9. doi: 10.1212/wnl.36.2.244.
- BackgroundBruyn G. Huntington's chorea: Historical clinical and laboratory synopsis. In: Vinken P, Bruyn G, eds. Handbook of Clinical Neurology. Amsterdam, 1968: 298-378.
- Background: PubMed 6225033Leigh RJ, Newman SA, Folstein SE, Lasker AG, Jensen BA. Abnormal ocular motor control in Huntington's disease. Neurology. 1983 Oct;33(10):1268-75. doi: 10.1212/wnl.33.10.1268.
- Background: PubMed 153122Caine ED, Hunt RD, Weingartner H, Ebert MH. Huntington's dementia. Clinical and neuropsychological features. Arch Gen Psychiatry. 1978 Mar;35(3):377-84. doi: 10.1001/archpsyc.1978.01770270127013.
- Background: PubMed 2524678Bamford KA, Caine ED, Kido DK, Plassche WM, Shoulson I. Clinical-pathologic correlation in Huntington's disease: a neuropsychological and computed tomography study. Neurology. 1989 Jun;39(6):796-801. doi: 10.1212/wnl.39.6.796.
- Background: PubMed 1479606Sorensen SA, Fenger K. Causes of death in patients with Huntington's disease and in unaffected first degree relatives. J Med Genet. 1992 Dec;29(12):911-4. doi: 10.1136/jmg.29.12.911.
Related trials for Huntington's Disease
RecruitingPhase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Huntington Disease
RecruitingPhase 3
Pridopidine Phase 3 Study in Huntington's Disease
Huntington Disease
Not yet recruiting
Creatine and Beta-Hydroxy-Beta-Methylbutyrate (HMB) Supplementation Plus Home-Based Exercise for Sarcopenia in Huntington's Disease
Huntington Disease, Sarcopenia
Recruiting
Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study
Caregivers
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merit Cudkowicz, MD MSc principal investigator
Massachusetts General Hospital
Michael McDermott, PhD principal investigator
University of Rochester, Biostatistics
Karl Kieburtz, MD MPH principal investigator
Director, Clinical Trials Coordination Center, University of Rochester
49 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing coenzyme Q10 in people with Huntington's Disease, run by Massachusetts General Hospital at 49 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 49 sites in United States, Canada, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Mar 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00608881, which also lists 76 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2016.