PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Start Date
1/4/2024
Completion Date
6/1/2030
Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Detailed Description
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. Regimens 1 and 2 are fully enrolled. 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. The trial sites listed below are actively recruiting eligible participants for Regimen 3. Regimen 3 is open label.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
ION717
Placebo
Conditions
Locations
Massachusetts General Hospital
Boston, Massachusetts 02114
United States
NYU Langone Health
New York, New York 10016
United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio 44106
United States
Royal Melbourne Hospital
Parkville, 3050
Australia
McGill University Health Centre
Montreal, Quebec H3A 2B4
Canada
Hôpital Universitaire Pitié Salpêtrière
Paris, 75013
France
University Medical Center Göttingen
Göttingen, 37075
Germany
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Israel
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, 20133
Italy
Mihara Memorial Hospital
Gunma, Isesaki-shi 372-0006
Japan
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi 187-8551
Japan
Neuromuscular Center Yoshimizu Hospital
Yamaguchi, Shimonoseki-shi 751-0826
Japan
Hospital Clinic De Barcelona
Barcelona, 08036
Spain