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PrP-targeting siRNA Safety & Mechanism Study
Sponsor: Broad Institute of MIT and Harvard
Summary
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
Official title: 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.
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2026-04
Completion Date
2029-08-14
Last Updated
2026-05-11
Healthy Volunteers
No
Conditions
Interventions
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
Locations (4)
Massachusetts General Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Columbia University Medical Center
New York, New York, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States