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PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 | Clinical Trials | Clareo Health

RECRUITINGPHASE1/PHASE2

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

A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease

NCT06153966•Ionis Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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, multi-center study in participants with prion disease. The study will consist of a screening period of up to 6 weeks, a 30-week treatment period, a 142-week open-label extension period and a 32-week post-treatment period. Multiple dose levels will be tested. The trial consists of three Regimens. Regimens 1 and 2 are fully enrolled. Participants in Regimens 1 and 2 received multiple doses of study drug (ION717 and placebo) during the 30-week double-blind treatment period; the order of doses (i.e. whether a given dose was ION717 or placebo) was blinded. The trial sites listed below are actively recruiting eligible participants for Regimen 3. Regimen 3 is open label.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

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.

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.

Locations (13)

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

McGill University Health Centre

Montreal, Quebec, Canada

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, Japan

Key Dates

Start Date

January 4, 2024

Primary Completion Date

February 1, 2027

Completion Date

June 1, 2030

Last Updated

March 19, 2026

Enrollment

ESTIMATED participants

Conditions

Prion Disease

Interventions

ION717

DRUG

Placebo

DRUG

Contacts

Ionis Pharmaceuticals, Inc.

CONTACT

(844) 892-5665 PrionDisease@clinicaltrialmedia.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 19, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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