Actively Recruiting
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-04-24
76
Participants Needed
13
Research Sites
334 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
CONDITIONS
Official Title
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of probable or definite prion disease
- Early-stage prion disease at screening
- Willingness to meet all study requirements including travel, procedures, measurements, and visits
- Has a caregiver aged 18 or older who can support and provide information during the trial
- Aged 18 years or older at the time of informed consent
You will not qualify if you...
- Clinically significant medical history, lab test, or physical exam abnormalities making participation unsuitable
- Contraindication or unwillingness to have an MRI
- Obstructive hydrocephalus, ventriculoperitoneal shunt, or implanted CNS catheter
- Known brain or spinal disease interfering with lumbar puncture, CSF circulation, or safety assessments
- Any other condition that may interfere with participation or study completion as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
2
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
3
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Actively Recruiting
4
Royal Melbourne Hospital
Parkville, Australia, 3050
Actively Recruiting
5
McGill University Health Centre
Montreal, Quebec, Canada, H3A 2B4
Active, Not Recruiting
6
Hôpital Universitaire Pitié Salpêtrière
Paris, France, 75013
Actively Recruiting
7
University Medical Center Göttingen
Göttingen, Germany, 37075
Actively Recruiting
8
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel, 6423906
Actively Recruiting
9
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Milan, Italy, 20133
Actively Recruiting
10
Mihara Memorial Hospital
Gunma, Isesaki-shi, Japan, 372-0006
Active, Not Recruiting
11
National Center of Neurology and Psychiatry
Tokyo, Kodaira-shi, Japan, 187-8551
Actively Recruiting
12
Neuromuscular Center Yoshimizu Hospital
Yamaguchi, Shimonoseki-shi, Japan, 751-0826
Actively Recruiting
13
Hospital Clinic De Barcelona
Barcelona, Spain, 08036
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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