Clinical Trials in Italy / NCT06153966
Active, not recruiting
Phase 1/2
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT06153966 · tracked via the Priya Life Science Italy tracker
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
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 |
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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 Italy