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Recruiting
NCT07444580
PrP-targeting siRNA Safety & Mechanism Study
Conditions: Prion Disease
Sex: All
Ages: 18 Years – N/A
Healthy volunteers: No
Phase: PHASE1
Enrollment: 30
Sponsor: Broad Institute of MIT and Harvard
Location: Massachusetts General Hospital Boston Massachusetts
Summary
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
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.
Source: ClinicalTrials.gov (NCT07444580). StuddyBuddy aggregates publicly available trial information.
