PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
NCT06153966 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 76
Last updated 2026-04-24
Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecal (IT) delivery of ION717.
Conditions
- Prion Disease
Interventions
- DRUG
-
ION717
ION717 will be administered by IT injection.
- DRUG
-
Placebo-matching ION717 will be administered by IT injection.
Sponsors & Collaborators
-
Ionis Pharmaceuticals, Inc.
lead INDUSTRY
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- DOUBLE
- Model
- PARALLEL
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-01-04
- Primary Completion
- 2027-02-28
- Completion
- 2030-06-30
- FDA Drug
- Yes
Countries
- United States
- Australia
- Canada
- France
- Germany
- Israel
- Italy
- Japan
- Spain
Study Locations
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