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PrP-targeting siRNA Safety & Mechanism Study

An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adult Patients Diagnosed With Symptomatic Prion Disease.

Who can join

Ages 18 and older · All sexes

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.

About the study

The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.

What is being tested

• PrP-siRNA (drug)

Sponsor: Broad Institute of MIT and Harvard · Participants: 30 · Started: May 15, 2026

Contact the study team

• Broad Institute · Phone: 617 714 7000

Official record on ClinicalTrials.gov — NCT07444580

Locations in the U.S.

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.