NCT07444580 PrP-targeting siRNA Safety & Mechanism Study
| NCT ID | NCT07444580 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Broad Institute of MIT and Harvard |
| Condition | Prion Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 45 participants |
| Start Date | 2026-05-15 |
| Primary Completion | 2029-08-14 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 45 participants in total. It began in 2026-05-15 with a primary completion date of 2029-08-14.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients and in pre-symptomatic adults at genetic risk for prion disease.
Eligibility Criteria
Key inclusion criteria for symptomatic patients: 1. clinically manifested symptoms of prion disease, in the opinion of the investigator 2. a diagnosis of probable prion disease according to CDC criteria 3. a positive CSF RT-QuIC or PRNP genetic test 4. no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15 5. availability of a caregiver to assist with study procedures Key inclusion criteria for pre-symptomatic patients: 1. 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 the investigator 2. a PRNP genetic test indicating the presence of a highly penetrant genetic variant causal for prion disease Key exclusion criteria for all arms: 1. pregnancy or unwillingness to comply with the contraceptive requirements during the study period 2. contraindication to lumbar puncture 3. recent participation in a different prion disease clinical trial Additional inclusion and exclusion criteria apply and will be evaluated at screening.
Contact & Investigator
Eric V Minikel, PhD
PRINCIPAL INVESTIGATOR
Broad Institute of MIT and Harvard
Frequently Asked Questions
Who can join the NCT07444580 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Prion Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07444580 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07444580 currently recruiting?
Yes, NCT07444580 is actively recruiting participants. Contact the research team at priontrials@broadinstitute.org for enrollment information.
Where is the NCT07444580 trial being conducted?
This trial is being conducted at Boston, United States, Rochester, United States, New York, United States, Cleveland, United States and 1 additional location.
Who is sponsoring the NCT07444580 clinical trial?
NCT07444580 is sponsored by Broad Institute of MIT and Harvard. The principal investigator is Eric V Minikel, PhD at Broad Institute of MIT and Harvard. The trial plans to enroll 45 participants.