This observational study will establish a clinical baseline and measure changes over time in movement, thinking, behavior, brain imaging, blood and spinal fluid markers in subjects with early stage Huntington's disease. Participants enrolled in this study may be eligible to participate in a future planned study of stem cell therapy for Huntington's Disease (HD). In-person study visits occur at screening, baseline, and every 6 months thereafter for a minimum of 12 months, with interim phone call assessments.
In PRE-CELL the investigators propose to enroll a cohort of early-stage HD patients in a prospective observational study designed to characterize clinical, neuro-imaging, laboratory and biomarker correlates of disease progression over 12-18 months. Subjects who complete a minimum of 12 months' participation in this trial will be candidates for enrollment in the future planned Phase 1 trial of intrastriatal delivery of mesenchymal stem cell (MSC)/Brain-derived neurotrophic factor (BDNF).
Study Type
OBSERVATIONAL
Enrollment
29
UC Davis Medical Center, Clinical Research Center
Sacramento, California, United States
Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan.
Time frame: Baseline and 12 or 18 months
Rate of change from baseline on the UHDRS total motor score
Time frame: Baseline and 12 or 18 months
Rate of change from baseline on the Total Functional Capacity score
Time frame: Baseline and 12 or 18 months
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