Prion disease is currently an invariably fatal condition without treatment or cure. New treatments are currently being investigated, and observational studies (clinical research with no drug treatment) are also being done to better understand the disease. This site will be regularly updated with available research study information for prion disease.
Below is a list of clinical research studies for prion diseases.
For additional information about each study, including eligibility criteria, study locations, and enrollment details, please visit the linked ClinicalTrials.gov pages.
Clinical Trials
Clinical Trial | ClinicalTrials.gov Page | Study Population | Status | Status | Community Statements |
|---|---|---|---|---|---|
| PrP-targeting siRNA Safety & Mechanism Study (PRiSM) | Early symptomatic prion disease and asymptomatic PRNP mutation carriers. | Recruiting | N/A | ||
| A Trial of ALN-6457 in Adult Participants With Symptomatic Prion Disease (PrD) | Early symptomatic prion disease patients. | Not Recruiting | N/A | Pending | |
| ION717-CS2: A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients with Prion Disease | Early symptomatic prion disease patients. | Not Recruiting | Not Recruiting | 3/17/2026 |
Observational Studies
Clinical Trial | ClinicalTrials.gov Page | Study Population | Status | Status |
|---|---|---|---|---|
| Teleneurology Assessment Program for Creutzfeldt-Jakob Disease (TAPCJD) | Symptomatic prion disease patients. | Recruiting | N/A | |
| PROSPECT Registry for Individuals at Risk for Genetic Prion Disease (PROSPECT) | Individuals at risk for prion disease. | Recruiting | N/A |