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, multi-center study in participants with prion disease. The study will consist of a screening period of up to 6 weeks, a 30-week treatment period, a 142-week open-label extension period and a 32-week post-treatment period. Multiple dose levels will be tested. The trial consists of three Regimens. Participants in Regimens 1 and 2 received multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses (i.e. whether a given dose was ION717 or placebo) was blinded. Regimen 3 is open label.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
85
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
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.
Royal Melbourne Hospital
Parkville, Australia
McGill University Health Centre
Montreal, Quebec, Canada
Hôpital Universitaire Pitié Salpêtrière
Paris, France
University Medical Center Göttingen
Göttingen, Germany
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, Italy
Mihara Memorial Hospital
Gunma, Isesaki-shi, Japan
...and 3 more locations