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

Recruiting now · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Prion Disease

In brief

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.

Key facts

Study ID

NCT06153966

Run by

Ionis Pharmaceuticals, Inc.

People needed

76

Starts

2024-01-04

Expected to finish

2030-06-01

Last updated by the study team

2026-08-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Hospital Clinic De Barcelona — Barcelona, Spain (enrolling)
• NYU Langone Health — New York, New York, United States (enrolling)
• Tel Aviv Sourasky Medical Center — Tel Aviv, Israel (enrolling)
• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta — Milan, Italy (enrolling)
• National Center of Neurology and Psychiatry — Tokyo, Kodaira-shi, Japan (enrolling)
• Neuromuscular Center Yoshimizu Hospital — Yamaguchi, Shimonoseki-shi, Japan (enrolling)
• Royal Melbourne Hospital — Parkville, Australia (enrolling)
• Hôpital Universitaire Pitié Salpêtrière — Paris, France (enrolling)
• University Medical Center Göttingen — Göttingen, Germany (enrolling)
• University Hospitals Cleveland Medical Center — Cleveland, Ohio, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• McGill University Health Centre — Montreal, Quebec, Canada
• Mihara Memorial Hospital — Gunma, Isesaki-shi, Japan

Full record on ClinicalTrials.gov

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