ION717: ION717 will be administered by IT injection.
Placebo: Placebo-matching ION717 will be administered by IT injection.
Study summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria
* A confirmed diagnosis of probable or definite prion disease.
* Early-stage prion disease at the time of Screening.
* Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits.
* Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial.
* Aged ≥ 18 at the time of informed consent.
Key Exclusion Criteria
* Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion.
* Any contraindication or unwillingness to undergo an MRI.
* Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter.
* Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment.
* Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.
Primary outcome measure(s)
Incidence of treatment-emergent adverse events. — Baseline up to Week 33
Trial sites (13)
Facility
City
Region
Status
Massachusetts General Hospital
Boston
Massachusetts
NYU Langone Health
New York
New York
University Hospitals Cleveland Medical Center
Cleveland
Ohio
Royal Melbourne Hospital
Parkville
Australia
McGill University Health Centre
Montreal
Quebec
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
National Center of Neurology and Psychiatry
Tokyo
Kodaira-shi
Neuromuscular Center Yoshimizu Hospital
Yamaguchi
Shimonoseki-shi
Hospital Clinic De Barcelona
Barcelona
Spain
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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