Actively Recruiting
PrP-targeting siRNA Safety & Mechanism Study
Led by Broad Institute of MIT and Harvard · Updated on 2026-05-11
30
Participants Needed
4
Research Sites
175 weeks
Total Duration
On this page
Sponsors
B
Broad Institute of MIT and Harvard
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
CONDITIONS
Official Title
PrP-targeting siRNA Safety & Mechanism Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Clinically manifested symptoms of prion disease as judged by the investigator
- Diagnosis of probable prion disease according to CDC criteria
- Positive cerebrospinal fluid RT-QuIC or PRNP genetic test
- No more than moderate functional impairment with an MRC-PDRS score of 15 or higher
- Availability of a study partner to assist with study procedures
You will not qualify if you...
- Pregnancy
- Contraindication to lumbar puncture (LP)
- Recent participation in a different prion disease clinical trial
- Additional inclusion and exclusion criteria will be evaluated at screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
2
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
3
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
4
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
Research Team
B
Broad Institute
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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