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 study will consist of a screening period of up to 6 weeks, a 30-week double-blind treatment period, a 70-week open-label extension period and a 32-week post-treatment period. During the double-blind period, the order of ION717 and placebo doses will be randomized and blinded to participants, study sites and the Sponsor. During the open-label extension period, all participants will receive ION717. Multiple dose levels will be tested.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
56
UCLA Neurology Clinic
Los Angeles, California, United States
University of Colorado Hospital
Denver, Colorado, United States
Incidence of treatment-emergent adverse events.
Time frame: Baseline up to Week 33
Maximum Observed Plasma Concentration (Cmax) of ION717
Time frame: on Day 1 and Week 9
Area Under the Plasma Concentration-time Curve (AUC) of ION717
Time frame: on Day 1 and Week 9
Half-life (t1/2λz) of ION717 in Plasma
Time frame: on Day 1 and Week 9
Cerebrospinal fluid (CSF) Concentration of ION717
Time frame: Pre-dose and at multiple points post-dose up to Week 33
Amount of ION717 Excreted in Urine
Time frame: Post-dose on Day 1
Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF
Time frame: Pre-dose and at multiple points post-dose up to Week 33
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Massachusetts General Hospital
Boston, Massachusetts, United States
NYU Langone Health
New York, New York, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Royal Melbourne Hospital
Parkville, Australia
University of Alberta
Edmonton, Alberta, Canada
McGill University Health Centre
Montreal, Quebec, Canada
Hôpital Universitaire Pitié Salpêtrière
Paris, France
University Medical Center Göttingen
Göttingen, Germany
...and 6 more locations