Prion Disease study: PrP-siRNA
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
- This study is testing whether PrP‑siRNA is safe and how it works in the body of people with prion disease or who carry a genetic risk.
- Who it's for
- You may be able to join if you have symptoms of prion disease or are an adult with a genetic risk for it.
- What's involved
- You will receive PrP‑siRNA, a small piece of genetic material, and have blood tests and check‑ups to see how safe it is.
- Time commitment
- The study lasts several months and includes multiple clinic visits for tests and monitoring.
- 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
- lack of clinically manifested symptoms of prion disease, as demonstrated by an MRC Score of 20, a MoCA score between 24-30 inclusive, and a...
You likely can't join if
- pregnancy or unwillingness to comply with the contraceptive requirements during the study period
- contraindication to lumbar puncture
- recent participation in a different prion disease clinical trial Additional inclusion and exclusion criteria apply and will be evaluated at screening.
See the full eligibility criteria
- 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
- lack of clinically manifested symptoms of prion disease, as demonstrated by an MRC Score of 20, a MoCA score between 24-30 inclusive, and a neurological exam with no clinically significant findings, in the opinion of...
- a PRNP genetic test indicating the presence of a highly penetrant genetic variant causal for prion disease Key
- pregnancy or unwillingness to comply with the contraceptive requirements during the study period
- contraindication to lumbar puncture
- recent participation in a different prion disease clinical trial Additional inclusion and exclusion criteria apply and will be evaluated at screening.
The study team makes the final eligibility decision.
Where it is enrolling
- 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, but 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?
This study is enrolling now at 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 enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.