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Tests treatment safety and results for Prion Disease
Official title PrP-targeting siRNA Safety & Mechanism Study
ClinicalTrials.gov ID: NCT07444580
What this study is testing
What is PrP-siRNA?
PrP-siRNA is an investigational medicine, being studied as a potential treatment for prion disease.
Also referred to as 2439-s4, 2439-exNA.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- clinically manifested symptoms of prion disease, in the opinion of the investigator;
- a diagnosis of probable prion disease according to CDC criteria;
- a positive CSF RT-QuIC or PRNP genetic test;
- no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15; and
- availability of a study partner to assist with study procedures. Key
You likely can't join if
- pregnancy;
- contraindication to LP; or
- recent participation in a different prion disease clinical trial. Additional inclusion and exclusion criteria apply and will be evaluated at...
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Cleveland, Ohio, United States
- Nashville, Tennessee, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States; Rochester, Minnesota, United States; New York, New York, United States; Cleveland, Ohio, United States; Nashville, Tennessee, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.