|Cohort||Population||Genomic DNA||RNA||Plasma||Serum||CSF||Whole Blood||Urine|
|PREDICTHD||HD gene/CAG-expansion positive||✔||✔||✔||✔||✔||✔|
- Study Subjects
Subjects completed annual study visits that included clinical assessment, MRI and biospecimen collection. Data can be requested through dbGaP.
- Available Biospecimens
DNA and RNA from blood, plasma, whole blood, CSF, and urine
The NINDS-supported PREDICT-HD study is a 32-site observational study of persons who have the gene expansion for Huntington Disease (HD), but do not have symptoms of disease warranting a diagnosis. The goals of the PREDICT-HD study involve laying the groundwork for the initiation of neuroprotective therapy in order to delay or conceivably prevent the onset of disease. The specific aims were designed to 1) improve prediction of disease diagnosis in healthy individuals using longitudinal measures of plasma, imaging, cognitive performances, motor ratings and psychiatric measures; 2) identify markers of disease progression prior to the clinical diagnosis and to characterize their natural history; and 3) improve the validity and reliability of disease measures upon which the power and sensitivity of multi-site trials and studies depend.
- Study Subjects
609 individuals participated in the study. Eligible subjects were required to have clinical symptoms of HD and a confirmatory family history of HD OR a CAG repeat expansion ≥ 36. Data can be requested through dbGaP.
2CARE is a 48 site, randomized, double-blind, placebo-controlled trial of coenzyme Q10 for the treatment of Huntington Disease (HD). The study enrolled 609 ambulatory subjects with HD who did not require 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).
Detailed study information can be found here.