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Clinical Trials in the USA / NCT06153966
Active, not recruiting Phase 1/Phase 2

PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

NCT06153966 · tracked via the Priya Life Science USA tracker
Sponsor
Ionis Pharmaceuticals, Inc.
Phase
Phase 1/Phase 2
Started
2024-01-04
Last updated
2026-09-15

Condition(s) studied

Prion Disease

Investigational drug(s) / intervention(s)

ION717Placebo

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)

Trial sites (13)

FacilityCityRegionStatus
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.

View NCT06153966 on ClinicalTrials.gov ↗ ← All trials in the USA