RecruitingPhase 1NCT07444580

PrP-targeting siRNA Safety & Mechanism Study

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.

Sponsor

Broad Institute of MIT and Harvard

Enrollment

30 participants

Start Date

May 15, 2026

Study Type

INTERVENTIONAL

Conditions

Prion Disease

Summary

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

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called PrP-siRNA for people with prion disease. The study is currently recruiting participants at 5 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGPrP-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(5)

Massachusetts General Hospital

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

Columbia University Medical Center

New York, New York, United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

View Full Details on ClinicalTrials.gov

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NCT07444580

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