Coenzyme Q10 in Huntington's Disease (HD)

NCT00608881 | Terminated Phase 3 Results DMC

• 3 other identifiers: 2CARE 01.005U01NS0525925R01NS052619
• Study Type: interventional
• Target: 609
• Locations: 3 countries
• Sites: 49

Brief 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.

Conditions

Huntington's Disease

Keywords

Huntington's disease; Huntington disease; HD; coenzyme Q10; CoQ

Outcome Measures

Primary Outcomes (1)

• 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).

  5 years

Secondary Outcomes (14)

• Change in Total Functional Capacity (TFC) Score From Baseline to Month 60

  Baseline and Month 60

• Change in Functional Checklist Score From Baseline to Month 60

  Baseline and Month 60

• Change in Independence Scale Score From Baseline to Month 60

  Baseline and Month 60

• Change in Total Motor Score From Baseline to Month 60

  Baseline and Month 60

• Change in Behavioral Frequency Score From Baseline to Month 60

  Baseline and Month 60

Study Arms (2)

A - coenzyme Q10 2400 mg/day

ACTIVE COMPARATOR

Randomized to active treatment (coenzyme Q10 2400 mg/day)

Drug: coenzyme Q10

B - Placebo

PLACEBO COMPARATOR

Randomized to placebo

Other: placebo

Interventions

coenzyme Q10 DRUG

4 - 300 mg CoQ chewable wafers taken orally twice a day

Also known as: CoQ

A - coenzyme Q10 2400 mg/day

placebo OTHER

an inactive substance

B - Placebo

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator
• University of Rochester: collaborator

Study Sites (49)

University of Alabama At Birmingham, Pediatric Neurology Childrens, Harbor Bldg Suite 314, 1600 7Th Avenue South

Birmingham, Alabama, 35233-1711, United States

Location

Mayo Clinic Arizona, 13400 East Shea Boulevard, Csu-Cp21B

Scottsdale, Arizona, 85259, United States

Location

WASHINGTON REGIONAL MEDICAL CENTER, 3215 N. North Hills Blvd

Fayetteville, Arkansas, 72703, United States

Location

University of California Irvine, Department of Neurology, 100 Irvine Hall

Irvine, California, 92697-4275, United States

Location

University of California Davis, Medical Center Dept of Neurology, Acc Building Suite 3700, 4860 Y Street

Sacramento, California, 95817, United States

Location

Colorado Neurological Institute, Movement Disorders Center, 701 East Hampden Avenue Suite 510

Littleton, Colorado, 80120, United States

Location

University of Florida Center for Movement Disorders and Neurorestoration, 3450 Hull Road, 4th Floor

Gainesville, Florida, 32607, United States

Location

UNIVERSITY OF MIAMI, 1150 NW 14th STREET, #401

Miami, Florida, 33136, United States

Location

University of South Florida, College of Medicine Dept of Neurology, 12901 Bruce B Downs Blvd Mdc-55

Tampa, Florida, 33612, United States

Location

Emory University, Wesley Woods Center, 1841 Clifton Road NE Room 314

Atlanta, Georgia, 30329, United States

Location

Idaho Elks Rehabilitation Hospital, 600 North Robbins Road

Boise, Idaho, 83702, United States

Location

Rush University Medical Center, Department of Neurological Sciences, 1725 West Harrison Suite 755

Chicago, Illinois, 60612, United States

Location

Indiana University School of Medicine, Outpatient Clinical Research Facility, 535 Barnhill Drive Room #150

Indianapolis, Indiana, 46202, United States

Location

University of Iowa Hospital and Clinics, 200 Hawkins Road, Room W263 General Hospital

Iowa City, Iowa, 52242-1000, United States

Location

University of Kansas Medical Center, Department of Neurology, 3599 Rainbow Blvd Mail Stop 2012

Kansas City, Kansas, 66160-7314, United States

Location

Hereditary Neurological Disease Centre (Hndc),3223 N. Webb, Suite 4

Wichita, Kansas, 67226, United States

Location

University of Maryland School of Medicine, 22 South Greene Street, N4 W49-B

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins University, 600 North Wolfe Street, Meyer 2-181

Baltimore, Maryland, 21287, United States

Location

Boston University School of Medicine, Department of Neurology, 715 Albany Street C329

Boston, Massachusetts, 02118, United States

Location

Massachusetts General Hospital, 149 13Th Street Suite 2241

Charlestown, Massachusetts, 02129, United States

Location

University of Michigan, 1500 E Medical Center Drive, B1 H202 Nuclear Medicine

Ann Arbor, Michigan, 48109-0028, United States

Location

Struthers Parkinson'S Center, 6701 Country Club Drive

Golden Valley, Minnesota, 55427, United States

Location

Washington University School of Medicine, Box 8111, 660 South Euclid

St Louis, Missouri, 63110, United States

Location

University of Las Vegas School of Medicine, 1707 W. Charleston Blvd, Suite 220

Las Vegas, Nevada, 89102, United States

Location

Cooper University Hospital

Camden, New Jersey, 08103, United States

Location

Nj Neuroscience Institute, Jfk Medical Center, 65 James Street

Edison, New Jersey, 08818, United States

Location

Albany Medical College, Parkinson'S Disease & Movement Disorders Ctr

Albany, New York, 12208, United States

Location

North Shore-Lij Health System, 350 Community Drive Room 110, Research Institute

Manhasset, New York, 11030, United States

Location

Columbia University, Sergievsky Center P&S Box 16, 630 West 168Th Street

New York, New York, 10032, United States

Location

University of Rochester, Department of Neurology, 919 Westfall Road Building C Suite 220

Rochester, New York, 14618, United States

Location

Duke University, 932 Morreene Road #213

Durham, North Carolina, 27705, United States

Location

Wake Forest University, Baptist Med Center, Department of Neurology, Medical Center Boulevard

Winston-Salem, North Carolina, 27157, United States

Location

University of Cincinnati/Cincinnati Children'S Hospital, 222 Piedmont Avenue, Suite 3200

Cincinnati, Ohio, 45219, United States

Location

OHIO STATE UNIVERSITY , 2006 Kenny Road

Columbus, Ohio, 43212, United States

Location

ST. LUKE'S HOSPITAL, 240 Centronia Road

Allentown, Pennsylvania, 18104, United States

Location

University of Pennsylvania, Pennsylvania Hospital Department of Neurology , 330 South 9Th Street

Philadelphia, Pennsylvania, 19107, United States

Location

University of Pittsburgh Kaufmann Medical Building, 3471 Fifth Avunue, Suite 811

Pittsburgh, Pennsylvania, 15213, United States

Location

BUTLER HOSPTIAL MOVEMENT DISORDER PROGRAM, 345 Blackstone Boulevard

Providence, Rhode Island, 02906, United States

Location

The University of Tennesee Health Science Cen, 855 Monroe Avenue, Department of Neurology, Room 415 Link Bldg

Memphis, Tennessee, 38163, United States

Location

UN oF TEXAS SOUTHWESTERN MED CENTER DALLAS, 5323 HARRY HINES BOULEVARD H1.108

Dallas, Texas, 75390-9016, United States

Location

Baylor College of Medicine, 6550 Fannin Suite 1801

Houston, Texas, 77030, United States

Location

Westmead Hospital, Department of Neurology Level 1, Po Box 533

Wentworthville, New South Wales, 2145, Australia

Location

University of Calgary, Heritage Medical Research Clinic, Trw Bldg 5 Floor, 3280 Hospital Dri. NW

Calgary, Alberta, T2N 4Z6, Canada

Location

University of Alberta, Glenrose Rehab Hosp, Movement Disorder Clinic , Rm 0601 Gleneast 10230 - 111 Avenue

Edmonton, Alberta, T5G 0B7, Canada

Location

Department of Medical Genetics, Ubc Hospital, Room S179-2211 Westbrook Mall

Vancouver, British Columbia, V6T 2B5, Canada

Location

London Health Sciences Centre, University Hospital, 339 Windermere Road

London, Ontario, N6A 5A5, Canada

Location

Centre For Movement Disorders, 2780 Bur Oak Avenue

Markham, Ontario, L4A 1G8, Canada

Location

NORTH YORK GENERAL HOSPITAL (2), 4001 Leslie Street

Toronto, Ontario, M2K 1E1, Canada

Location

North York General Hospital, 4001 Leslie Street

Toronto, Ontario, M2R 1N5, Canada

Location

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MeSH Terms

Conditions

Huntington Disease

Interventions

coenzyme Q10

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Limitations and Caveats

An interim analysis for futility revealed a conditional power of \< 5% for the primary analysis, and the trial was halted in July, 2014. Only data collected prior to this time were included in the final analyses.

Results Point of Contact

• Title: Dr. Merit Cudkowicz
• Organization: Massachusetts General Hospital

mcudkowicz@partners.org

617-726-0813

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Julieanne Dorn Professor of Neurology

Study Record Dates

• First Submitted: February 4, 2008
• First Posted: February 6, 2008
• Study Start: March 1, 2008
• Primary Completion: November 1, 2014
• Study Completion: May 1, 2015
• Last Updated: March 30, 2016
• Results First Posted: March 30, 2016

Record last verified: 2016-02

Locations

US (41); AU (1); CA (7)