PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
- Registration Number
- NCT06153966
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief 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, 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 56
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ION717 + Placebo, Regimen 1 ION717 Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period. ION717 + Placebo, Regimen 1 Placebo Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period. ION717 + Placebo, Regimen 2 ION717 Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period. ION717 + Placebo, Regimen 2 Placebo Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 70-week open-label extension period.
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events. Baseline up to Week 33
- Secondary Outcome Measures
Name Time Method Maximum Observed Plasma Concentration (Cmax) of ION717 on Day 1 and Week 9 Area Under the Plasma Concentration-time Curve (AUC) of ION717 on Day 1 and Week 9 Half-life (t1/2λz) of ION717 in Plasma on Day 1 and Week 9 Cerebrospinal fluid (CSF) Concentration of ION717 Pre-dose and at multiple points post-dose up to Week 33 Amount of ION717 Excreted in Urine Post-dose on Day 1 Percent Change from Baseline in Prion Protein (PrP) Concentration in CSF Pre-dose and at multiple points post-dose up to Week 33
Trial Locations
- Locations (16)
UCLA Neurology Clinic
🇺🇸Los Angeles, California, United States
University of Colorado Hospital
🇺🇸Denver, Colorado, United States
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
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
McGill University Health Centre
🇨🇦Montréal, Quebec, Canada
Hôpital Universitaire Pitié Salpêtrière
🇫🇷Paris, France
University Medical Center Göttingen
🇩🇪Göttingen, Germany