NCT06153966

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 76 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Prion Disease

Interventions

Type	Name	Description
DRUG	ION717	ION717 will be administered by IT injection.
DRUG	Placebo	Placebo-matching ION717 will be administered by IT injection.

Timeline

• Start date: 2024-01-04
• Primary completion: 2027-02-01
• Completion: 2030-06-01
• First posted: 2023-12-01
• Last updated: 2026-04-13

Locations

13 sites across 9 countries: United States, Australia, Canada, France, Germany, Israel, Italy, Japan, Spain

Regulatory

• FDA-regulated drug study