Prion Disease is currently an invariably fatal condition without treatment or cure. Despite multiple prior treatment studies, including quinacrine, doxycycline, and pentosan polysulfate, no investigational treatments have benefited patients with prion disease. However, new treatments are currently being investigated. This site will be regularly updated with available treatment trial information for prion disease.
Below is a list of clinical trials 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 Trial | ClinicalTrials.gov Page | Enrollment (U.S. Sites) | Enrollment (International Sites) | Community Statement |
|---|---|---|---|---|
| ION717-CS2: A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients with Prion Disease | Closed | Open | ||
| PrP-targeting siRNA Safety & Mechanism Study (PRiSM) | Open | N/A |