U.S., March 4 -- ClinicalTrials.gov registry received information related to the study (NCT07444580) titled 'First-in-human Trial of PrP-siRNA in Symptomatic Prion Disease.' on Feb. 24.
Brief Summary: The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Study Start Date: April, 2026
Study Type: INTERVENTIONAL
Condition:
Prion Disease
Intervention:
DRUG: PrP-siRNA
Intrathecally administered divalent siRNA designed to target the PRNP mRNA. The structure has been published in DOI: 10.1101/2024.12.05.627039
Recruitment Status: NOT_YET_RECRUITING
Sponsor: Broad Institute of MIT and Harvard
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