Ongoing Treatment Trials for Prion Disease

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. 

This is a first-in-human, randomized, multicenter study in participants with prion disease. Eligible participants will receive ION717 and a placebo. The order of doses will be randomized and blinded to participants, study sites, and the Sponsor. The study will consist of a screening period of up to 6 weeks, a 24-week double-blind treatment period, and a 36-week post-treatment period. Multiple dose levels will be tested.

Please click here for the complete study details on the page.