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.
Broad Institute of MIT and Harvard
30 participants
Apr 1, 2026
INTERVENTIONAL
Conditions
Summary
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Eligibility
Inclusion Criteria5
- 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 Criteria3
- pregnancy;
- contraindication to LP; or
- recent participation in a different prion disease clinical trial.
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Interventions
Intrathecally administered divalent siRNA designed to target the PRNP mRNA. The structure has been published in DOI: 10.1101/2024.12.05.627039
Locations(3)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT07444580