PrProfile (NCT06153966): ION717 in prion disease

Key facts

• Status: Active, not recruiting (enrolment completed December 2024).
• Who can join: Symptomatic prion disease (details not read)
• Where: North American and Japanese sites on ClinicalTrials.gov (see Registry line). The official EU CTIS record 2023-503355-98-00 adds one site in each of four EU member states: Universitaetsmedizin Goettingen, Fondazione IRCCS Istituto Neurologico Carlo Besta in Milan, Hospital Clinic de Barcelona and Assistance Publique Hopitaux de Paris, with Italy as reference member state and status shown as authorised. It gives a recruitment count of 2 per EU state (8 in the EU) and no planned total for the whole trial.
• Sponsor: Ionis Pharmaceuticals
• Dates: Primary completion estimated February 2027, study completion estimated June 2030, registry last updated 17 August 2026 with actual enrolment 85 after regimen 3 started
• Registry: Phase 1/2a, start 2024-01-04, 13 locations (Massachusetts, New York, Ohio, Quebec, and Isesaki, Kodaira, Shimonoseki in Japan); registry status active not recruiting; EudraCT 2023-503355-98; planned participants 76.
• On 2026-03-17 Ionis re-opened the trial with a third dosing regimen; 56 symptomatic patients had enrolled in 2024 across 16 sites; only Cleveland, Tokyo and Yamaguchi were active; pre-symptomatic carriers are not eligible.
• The NPDPSC studies list still shows not recruiting; sources disagree, so confirm with the site.
• 2026-10-15 Correction C-007: PrProfile status is unclear: registry says active not recruiting; sponsor re-opened a third regimen in March 2026; NPDPSC list says not recruiting.

Timeline

1. 2024Ionis announces the early trial fully enrolled.
2. 2026Ionis re-opens the trial with a third dosing regimen (three sites active).