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PrP-targeting siRNA Safety & Mechanism Study

NCT07444580 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-05-11

No results posted yet for this study

Summary

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

Conditions

  • Prion Disease

Interventions

DRUG

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

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Broad Institute of MIT and Harvard

    lead OTHER

Principal Investigators

  • Eric V Minikel, PhD · Broad Institute of MIT and Harvard

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-30
Primary Completion
2029-08-14
Completion
2029-08-14
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07444580 on ClinicalTrials.gov