RecruitingPhase 1

PrP-targeting siRNA Safety & Mechanism Study

United States30 participantsStarted 2026-04

Plain-language summary

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

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. 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; and
✓. availability of a study partner to assist with study procedures.

Exclusion criteria

✕. pregnancy;
✕. contraindication to LP; or
✕. recent participation in a different prion disease clinical trial.

What they're measuring

Frequency of adverse events

Timeframe: Baseline to week 24

Trial details

NCT IDNCT07444580

SponsorBroad Institute of MIT and Harvard

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2029-08-14

Contact for this trial

Broad Institute

617 714 7000 priontrials@broadinstitute.org

View on ClinicalTrials.gov More Prion Disease trials