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

This is a multi-center, randomized, double-blind, placebo-controlled trial of CoQ involving 609 ambulatory subjects with HD who are not requiring skilled care or institutionalization. Eligible subjects were randomized to receive either CoQ 2400 mg/day or matching placebo. Subjects were followed prospectively and systematically for 60 months of double-blind observation. The primary outcome variable is a combination of time to death (for subjects who die) and the change from baseline to Month 60 in Total Functional Capacity (TFC) score (for subjects who survive). The primary hypothesis of the study is that chronic treatment of early-stage HD subjects with high-dosage CoQ will slow the progressive functional decline of HD.

• Study Design:
  • Clinical Trial
• Study Type:
  • Multicenter
  • Randomized
  • Double-Blind
  • Placebo-Controlled
  • Clinical Trial
• Total number of consented subjects: 609
• Subject Sample Telemetry Report (SSTR)

Inclusion Criteria

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

1. Subjects must have clinical features of HD, and a confirmatory family history of HD OR a CAG repeat expansion >/= 36.
2. TFC > 9 (as rated by the Investigator during the Baseline Visit PRIOR to randomization).
3. Must be ambulatory and not require skilled nursing care.
4. Age >/= 16 years.
5. Women must not be able to become pregnant (e.g., are 2 years post-menopausal, surgically sterile (hysterectomy or tubal ligation) or using adequate birth control methods for the duration of the study). Adequate birth control includes: abstinence; oral, implanted or injected contraceptives, e.g., birth control pills; intra-uterine device; barrier (e.g., vaginal ring or diaphragm/cervical cap with spermicide; transdermal patch and/or partner vasectomy). Reliable contraception must have been in use 60 days prior to the Baseline Visit.
6. 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. Any changes to these medications mandated by clinical conditions will be systematically recorded and the subject will be permitted to remain in the trial.
7. Able to give informed consent and comply with trial procedures (for minor subjects under the age of 18, both parental permission and child assent required).
8. Able to take oral medication.
9. Able 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. While not required to attend all study visits, the caregiver/informant should attend the Screening and Baseline Visits to participate in the informed consent process and to receive study information and instructions; at the investigator's discretion, independently high functioning individuals may be permitted to enroll without a caregiver.
10. 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. (For minor subjects, age < 18, the subject's parent or legal guardian is the responsible person.)

Exclusion Criteria

1. History or known sensitivity of intolerability to CoQ.
2. Exposure to any investigational agent within 30 days of the Baseline visit.
3. Clinical evidence of unstable medical illness in the investigator's judgment.
4. Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression or suicidal ideation within 90 days of the Baseline visit.
5. Substance (alcohol or drug) abuse within one year of the Baseline visit.
6. Women who are pregnant or breastfeeding.
7. Use of supplemental coenzyme Q10 within 30 days prior to the Baseline visit.
8. Clinically serious abnormalities in the screening laboratory studies (Screening creatinine greater than 2.0mg/dL, alanine aminotransferase (ALT) or total bilirubin greater than 3 times the upper limit of normal, absolute neutrophil count of </= 1000/μl, platelet concentration of < 100,000/μl, hematocrit level of < 33 for female or < 35 for male, or coagulation tests > 1.5 times upper limit of normal).
9. Known allergy to the color additive FD and C #5 yellow lake (also known as tartrazine) or any other ingredient in the study drug (active and placebo).

2CARE data were recorded via the eClinical System, utilizing a suite of applications, including Data Standard Management (eDictionary), Clinical Data Management (eData Management), Clinical Trials Administration (eStudy Conduct), Electronic Data Capture (Data Capture) and Collaborative Study Portals.

• Primary Phenotype: Huntington Disease

• HTT

• BioProject
• MeSH
• Gene
• Clinical Trials
  • NCT00608881

• Principal Investigators
  • Merit E. Cudkowicz MD MSc. Massachusetts General Hospital, Boston, MA, USA.
  • Michael P. McDermott, PhD. University of Rochester, Rochester, NY, USA.
• Funding Sources
  • R01 NS052592. National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
  • R01 NS052619. National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.