Clinical trialNCT07444580RecruitingPhase 1Interventional

PrP-targeting siRNA Safety & Mechanism Study

SponsorBroad Institute of MIT and HarvardEnrollment30Prion Disease

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Eric V Minikel, PhD
Broad Institute of MIT and Harvard
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

5 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Massachusetts General Hospital
Boston, Massachusetts, United States
Steven E Arnold, MD
Recruiting
Mayo Clinic
Rochester, Minnesota, United States
Vijay K Ramanan, MD PhD
Recruiting
Columbia University Medical Center
New York, New York, United States
Lawrence Honig, MD PhD
Recruiting
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Brian S Appleby, MD
Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Amanda Peltier, MD
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

PrP-siRNA

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 15 May 2026
Primary completion: 14 Aug 2029
Completion: 14 Aug 2029
Last update posted: 5 Jun 2026
Registered enrollment: 30
Registry id: NCT07444580

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.