PrP-targeting siRNA Safety & Mechanism Study
Key facts
- Where
- Boston, Cleveland, Nashville, New York, and Rochester, United StatesSee the 5 sites
- Phase
- Phase 1
Where in United States
- Massachusetts General HospitalBoston, United StatesContact: MGH Prion Studies CoordinatorTaking people
- University Hospitals Cleveland Medical Center*Cleveland, United StatesContact: Mary Andrews, BA, RRT, CCRPTaking people
- Vanderbilt University Medical CenterNashville, United StatesContact: Kelly Harper, BPSTaking people
- Columbia University Medical CenterNew York, United StatesContact: Katrina Cuasay, BATaking people
- Mayo ClinicRochester, United StatesContact: Sherry Klingerman, CCRPTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Broad Institute of MIT and Harvard
- Trial code
- NCT07444580
- Record updated
- 16 October 2026
- Original description
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.Read full original descriptionShow less
An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adults Diagnosed With Symptomatic Prion Disease and in Pre-symptomatic Adults at Genetic Risk for Prion Disease.
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: 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; lack of clinically manifested symptoms of prion disease, as demonstrated by an MRC Score of 20, a MoCA score between 24-30 inclusive, and a neurological exam with no clinically significant findings, in the opinion of the investigator; a PRNP genetic test indicating the presence of a highly penetrant genetic variant causal for prion disease.
Exclusion: recent participation in a different prion disease clinical trial; Additional inclusion and exclusion criteria apply and will be evaluated at screening; pregnancy or unwillingness to comply with the contraceptive requirements during the study period; contraindication to lumbar puncture.