NCT07444580PHASE1Recruiting

PrP-targeting siRNA Safety & Mechanism Study

Broad Institute of MIT and Harvard

Start Date

5/15/2026

Completion Date

8/14/2029

Summary

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

Detailed Description

This is a first-in-human, open label, single ascending dose study in participants with prion disease. The study will consist of a screening period of up to 2 weeks, administration of a single intrathecal dose of PrP-siRNA, and a 24-week follow-up period. Multiple dose levels will be tested. This trial also includes an observational arm in which participants will not receive investigational drug, and will be followed for an 8-week period after baseline.

Eligibility Criteria

Age Range: 18 years to No maximum

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.

Interventions

DRUG

PrP-siRNA

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Conditions

Prion Disease

Locations

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Columbia University Medical Center

New York, New York 10032

United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio 44106

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States