A Pre-Cellular Therapy Observational Study in Early Huntington's Disease (PRE-CELL)
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.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||A Pre-Cellular Therapy Observational Study in Early Huntington's Disease|
- Rate of change from baseline in white matter volume on magnetic resonance imaging (MRI) brain scan. [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]
- Rate of change from baseline on the UHDRS total motor score [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]
- Rate of change from baseline on the Total Functional Capacity score [ Time Frame: Baseline and 12 or 18 months ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples Without DNA
Safety labs: Complete blood count, comprehensive metabolic panel, international normalized ration, partial thromboplastic time, thyroid stimulating hormone, urinalysis, HIV screen.
Biomarkers: Brain-derived neurotrophic factor (BDNF) in cerebrospinal fluid (CSF) and Plasma, BDNF related and Huntington's Disease (HD) specific gene transcription markers in CSF and blood, Small molecule markers of HD in CSF and plasma
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
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).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01937923
|United States, California|
|UC Davis Medical Center, Clinical Research Center|
|Sacramento, California, United States, 95817|
|Principal Investigator:||Vicki Wheelock, MD||University of California, Davis|