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PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Sponsor: Ionis Pharmaceuticals, Inc.
Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Official title: A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
76
Start Date
2024-01-04
Completion Date
2030-06
Last Updated
2026-04-09
Healthy Volunteers
No
Conditions
Interventions
ION717
ION717 will be administered by IT injection.
Placebo
Placebo-matching ION717 will be administered by IT injection.
Locations (13)
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
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
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan
Neuromuscular Center Yoshimizu Hospital
Yamaguchi, Shimonoseki-shi, Japan
Hospital Clinic De Barcelona
Barcelona, Spain