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Study of Safety and Effects of Single Intrathecal Dose of PrP-siRNA in Adults with Symptomatic Prion Disease
Led by Broad Institute of MIT and Harvard · Updated on 2026-06-05
30
Participants Needed
5
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying the safety, tolerability, and how the body processes and responds to PrP-siRNA in adults with symptomatic prion disease. This is a first-of-its-kind, open-label trial focusing on patients diagnosed based on clinical symptoms and specific tests. The study aims to evaluate different doses to understand their effects on prion disease markers and safety outcomes. Participants will be divided into two groups. One group will receive a single intrathecal dose of PrP-siRNA at ascending doses of 50, 100, or 200 mg and will be monitored closely for 24 weeks through outpatient visits after initial hospitalization. The other group will be observational, undergoing lumbar punctures and assessments without receiving the drug and will be followed for 8 weeks. During the study, participants will undergo lumbar punctures, safety monitoring, and evaluations of prion protein concentrations in cerebrospinal fluid and plasma at multiple timepoints up to 24 weeks after dosing. Researchers will track adverse events and drug levels to assess safety and pharmacodynamics. The total participation time varies by group, lasting up to 24 weeks for treated participants and 8 weeks for observational participants.
CONDITIONS
Brief Title
PrP-targeting siRNA Safety & Mechanism Study
Research Team
B
Broad Institute
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