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NCT06153966PHASE1, PHASE2Recruiting

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

Ionis Pharmaceuticals, Inc.

Start Date

1/4/2024

Completion Date

6/1/2030

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 Criteria

Age Range: 18 years to No maximum

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.

Interventions

DRUG

ION717

DRUG

Placebo

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Conditions

Prion Disease

Locations

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

NYU Langone Health

New York, New York 10016

United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio 44106

United States

Royal Melbourne Hospital

Parkville, 3050

Australia

McGill University Health Centre

Montreal, Quebec H3A 2B4

Canada

Hôpital Universitaire Pitié Salpêtrière

Paris, 75013

France

University Medical Center Göttingen

Göttingen, 37075

Germany

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906

Israel

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Milan, 20133

Italy

Mihara Memorial Hospital

Gunma, Isesaki-shi 372-0006

Japan

National Center of Neurology and Psychiatry

Tokyo, Kodaira-shi 187-8551

Japan

Neuromuscular Center Yoshimizu Hospital

Yamaguchi, Shimonoseki-shi 751-0826

Japan

Hospital Clinic De Barcelona

Barcelona, 08036

Spain