Phase 12a Study to Assess Safety and Effects of Intrathecal ION717 in Adults with Prion Disease

What this Trial Is About

Researchers are studying the safety, tolerability, pharmacokinetics, and pharmacodynamics of ION717, delivered through intrathecal injection, in people with prion disease. This is a first-in-human, multi-center study that includes participants with early-stage prion disease. The trial aims to understand how the body handles ION717 and how the drug affects prion protein levels. The study includes three regimens. Regimens 1 and 2 involve a 30-week double-blind treatment period where participants receive multiple doses of ION717 and placebo in a blinded order, followed by a 142-week open-label extension where all participants receive ION717. Regimen 3 is an open-label phase during which participants receive multiple doses of ION717 throughout both the 30-week treatment and 142-week extension periods. The study also includes a 32-week post-treatment follow-up. Participants will be involved for up to about 5 years, including screening, treatment, extension, and post-treatment periods. Assessments include monitoring for adverse events, measuring drug levels in plasma and cerebrospinal fluid, urine analysis, and tracking changes in prion protein concentrations. Safety and tolerability are closely monitored throughout the study, particularly during the initial 33 weeks. The study collects detailed pharmacokinetic and pharmacodynamic data to evaluate the effects of ION717.

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717