Status: Recruiting
PrP-targeting siRNA Safety & Mechanism Study
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Participants needed: 30
Trial details
Phase: Phase 1Age: 18+Biological sex: AllType: InterventionalSponsor: Broad Institute of MIT and HarvardUpdated: Jun 5, 2026Locations: 5
Eligibility criteria
clinically manifested symptoms of prion disease, in the opinion of the investiga... [+4]
pregnancy; [+2]