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Clinical trial

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.

NCT07444580 is registered in ClinicalTrials.gov with Broad Institute of MIT and Harvard as lead sponsor.

NCT NCT07444580
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Registry record

As filed in ClinicalTrials.gov.

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.
Lead sponsor
Broad Institute of MIT and Harvard
Phase
Phase 1
Overall status
Recruiting
Start date
May 15, 2026
Completion date
August 14, 2029
Enrollment
30 (estimated)
Sites
5

Conditions

As the registry lists them; a condition with its own page is linked.

prion disease