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- Study Description
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 Types: Multicenter, Randomized, Double-Blind, Placebo-Controlled, Clinical Trial
Number of study subjects that have individual level data available through Authorized Access: 609
- Authorized Access
- Publicly Available Data (Public ftp)
Connect to the public download site. The site contains release notes and manifests. If available, the site also contains data dictionaries, variable summaries, documents, and truncated analyses.
- Study Inclusion/Exclusion 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 confirmatory family history of HD OR a CAG repeat expansion >/= 36.
- TFC > 9 (as rated by the Investigator during the Baseline Visit PRIOR to randomization).
- Must be ambulatory and not require skilled nursing care.
- Age >/= 16 years.
- 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.
- 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.
- 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).
- Able to take oral medication.
- 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.
- 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.)
- History or known sensitivity of intolerability to CoQ.
- Exposure to any investigational agent 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 2.0mg/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).
- 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).
- Study History
Phase Completion Date Activity Planning 9/27/2005 Project award granted 11/9/2006 Investigator/Coordinator Orientation Meeting 8/31/2007 Receipt of FDA non-hold letter 12/3/2007 Canadian approval of Clinical Trial Application; amendment #3 approved 5/26/2008 2/20/2008 Delivery of drug supply to drug packaging unit (Clinical Materials Services Unit) 3/7/2008 First authorization granted for drug distribution to approved sites 3/12/2008 Database completed Implementation 3/19/2008 Begin screening 4/9/2008 First enrollment 4/16/2008 Distribution of sub-contracts to sites 8/29/2011 Interim analysis for futility 6/25/2012 Completion of enrollment 8/2/2012 Interim analysis for futility 7/2014 Interim analyses for futility and efficacy (unscheduled) 11/2014 Completion of follow-up of subjects Analysis/Publication 4/17/2015 Secure database 5/18/2015 Lock database 7/10/2015 Unblinding and presentation of results to Steering Committee; review of draft manuscript 7/30/2015 Presentation of results to investigators and coordinators
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.
- Selected publications
- Diseases/Traits Related to Study (MESH terms)
- Primary Phenotype: Huntington Disease
- Links to Related Resources
- Authorized Data Access Requests
- Study Attribution