NCT07444580
PrP-targeting siRNA Safety & Mechanism Study
Recruiting · Phase 1 · Broad Institute of MIT and Harvard · registry updated 2026-10-16
Inclusion and exclusion lines below are quoted from ClinicalTrials.gov. No match score is shown, because a score needs a person's age, biomarkers, and treatment dates. Confirm the record with the study team.
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients and in pre-symptomatic adults at genetic risk for prion disease.
Inclusion
- inclusion criteria for symptomatic patients:
- clinically manifested symptoms of prion disease, in the opinion of the investigator
- a diagnosis of probable prion disease according to CDC criteria
- a positive CSF RT-QuIC or PRNP genetic test
- no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15
- availability of a caregiver to assist with study procedures
Exclusion
- exclusion criteria for all arms:
- pregnancy or unwillingness to comply with the contraceptive requirements during the study period
- contraindication to lumbar puncture
- recent participation in a different prion disease clinical trial
- Additional inclusion and exclusion criteria apply and will be evaluated at screening.