NCT07444580 · Phase 1 · RECRUITING
PrP-targeting siRNA Safety & Mechanism Study
This trial is testing a gene-silencing drug called PrP-siRNA, which is designed to reduce production of the prion protein that drives prion disease. Researchers are measuring safety, tolerability, and whether the drug actually lowers prion protein levels in the body. It is a Phase 1 trial, meaning the primary goal is establishing that it is safe enough to study further — it is not yet proven or approved for any use.
Eligibility criteria
Key inclusion criteria: 1. clinically manifested symptoms of prion disease, in the opinion of the investigator; 2. a diagnosis of probable prion disease according to CDC criteria; 3. a positive CSF RT-QuIC or PRNP genetic test; 4. no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15; and 5. availability of a study partner to assist with study procedures. Key exclusion criteria: 1. pregnancy; 2. contraindication to LP; or 3. recent participation in a different prion disease clinical trial. Additional inclusion and exclusion criteria apply and will be evaluated at screening.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-06-05