PrP-targeting siRNA Safety & Mechanism Study

ClinicalTrials.gov ID: NCT07444580

Public ClinicalTrials.gov record NCT07444580. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Oct 23, 2026, 5:30 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered PrP-siRNA in Adults Diagnosed With Symptomatic Prion Disease and in Pre-symptomatic Adults at Genetic Risk for Prion Disease.

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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

Study identification

NCT ID: NCT07444580
Recruitment status: Recruiting
Study type: Interventional
Phase: Phase 1
Lead sponsor: Broad Institute of MIT and Harvard; Other
Enrollment: 45 participants

Conditions and interventions

Conditions

Prion Disease

Interventions

PrP-siRNA Drug

Drug

Eligibility (public fields only)

Age range: 18 Years and older
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: May 15, 2026 (Actual)
Primary completion: Aug 14, 2029 (Estimated)
Completion: Aug 14, 2029 (Estimated)
Last update posted: Oct 16, 2026

2026 – 2029

United States locations

U.S. sites: 5
U.S. states: 5
U.S. cities: 5

Facility	City	State	ZIP	Site status
Massachusetts General Hospital	Boston	Massachusetts	02114	Recruiting
Mayo Clinic	Rochester	Minnesota	55905	Recruiting
Columbia University Medical Center	New York	New York	10032	Recruiting
University Hospitals Cleveland Medical Center	Cleveland	Ohio	44106	Recruiting
Vanderbilt University Medical Center	Nashville	Tennessee	37232	Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT07444580, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Oct 16, 2026 · Synced Oct 23, 2026

Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT07444580 live on ClinicalTrials.gov.

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