Coenzyme Q10 (CoQ) in Huntington[Single Quote]s Disease (HD) (2CARE)

Study ID
STU 032011-013

Cancer Related
No

Healthy Volunteers
No

Study Sites

Contact
Holly Lawrence
214-648-9312
holly.lawrence@utsouthwestern.edu

Principal Investigator
Padraig O'Suilleabhain

Summary

The 2CaRe trial was designed in the model of the [Quote]large simple trial[Quote]. only the most relevant data will be collected, with visits at six-month intervals that are short enough to allow capture of important information regarding subject safety and change over time and to maintain subject retention, but long enough to minimize data collection. The Researchers will collect the following information: medical history, demographics (birth date, ethnicity, gender, highest level of education, occupation and marital status), vital sign measurements (pulse, blood pressure, weight and height) results of laboratory tests, concomitant medications, adverse events, questionnaires, findings from physical and neurological exams, results of pregnancy tests and rating scales used to measure status and progression of HD. interim analysis as part of the futility design of the study may lead to premature modification or stopping of the trial if evidence emerges that CoQ is either clearly ineffective or clearly efficacious in terms of slowing functional decline in HD. Secondary analyses will be done to determine if CoQ is more effective with very long expansion or with smaller expansion.

This is a multi-center, randomized, double-blind, placebo-controlled study which will include approximately 608 ambulatory subjects with early HD (aged 16 or older), who are not requiring skilled care or institutionalization, and who meet eligibility criteria (specified below) at approximately 46 Huntington Study Group investigative sites in north america and australia. about 40 people (2 children and 38 adults) will take part in this study at uT Southwestern in order to complete the study with 20 subjects. This study is also taking place at a number of other medical facilities around the country and in Canada and australia. There will be a total of 608 people participating in this research study throughout the united States, Canada, and australia. Subjects will be randomized to one of the two treatment arms, either CoQ 2400 mg/day or matching placebo with 1:1 allocation.

Subjects will participate in the study for approximately 5 years depending on their time of enrollment with visits in 6 month intervals.

Participant Eligibility

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

* Subjects must have been diagnosed with HD with clinical features of HD and a confirmatory family history of HD, OR a CAG repeat expansion >= 36.

* TFC >= 9

* Must be ambulatory and not require skilled nursing care

* Males and females 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.

* 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.. 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.

* 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.

* A designated individual will be identified by the subject to participate in the ongoing consent process should the subject[Single Quote]s cognitive capacity to consent become compromised during participation in the study. (For minor subjects, age <18, the subject[Single Quote]s parent or legal guardian is the responsible person.)

Spanish speaking subjects will be eligible to participate.