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RECRUITING
NCT06153966
PHASE1/PHASE2

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

Sponsor: 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.

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

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

76

Start Date

2024-01-04

Completion Date

2030-06

Last Updated

2026-04-09

Healthy Volunteers

No

Conditions

Interventions

DRUG

ION717

ION717 will be administered by IT injection.

DRUG

Placebo

Placebo-matching ION717 will be administered by IT injection.

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

National Center of Neurology and Psychiatry

Tokyo, Kodaira-shi, Japan

Neuromuscular Center Yoshimizu Hospital

Yamaguchi, Shimonoseki-shi, Japan

Hospital Clinic De Barcelona

Barcelona, Spain