Familial Fatal Insomnia Clinical Trial
Official title:
Biomarker Profiling in Individuals at Risk for Prion Disease
We are doing this research to identify biomarkers in individuals who are at-risk for familial prion disease. We hope to use these biomarkers to predict timing of disease onset in pre-symptomatic individuals and to guide the direction of future clinical trials.
This study aims to measure biomarkers longitudinally in individuals at risk of developing genetic prion disease to identify clinical assays and molecular markers that: can inform our understanding of pre-clinical pathology, predict timing of disease onset in pre-symptomatic individuals, and enable development and evaluation of novel treatment efficacy in pre-symptomatic or early symptomatic individuals. Participation in the study involves annual visits to the clinic site in Charlestown, MA. Study visits include: a medical exam, blood draws, cognitive tests and questionnaires, spinal fluid collection, and (optional) MRI. Travel support and stipend is provided for interested individuals. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04846335 -
Familiar Fatal Insomnia: Preventive Treatment With Doxycycline in Subject With Disease Risk
|
Phase 2 |