Prion Disease Clinical Trial
Official title:
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
This is a first-in-human, randomized, multi-center study in participants with prion disease. Eligible participants will receive ION717 and 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. As of April 2024, study recruitment is paused because capacity has been reached for the current stage of the study. Recruitment is anticipated to resume later in 2024. Central Contact: Ionis Pharmaceuticals Telephone: (844) 221-3587 Email: PrionDisease@clinicaltrialmedia.com ;
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