Clinical Trial

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Study acronym: HORIZON
Recruiting Phase 1
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Summary
The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA). The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).
Protocol Amendment History 31 amendments
This ClinicalTrials.gov record has been amended 31 times since 2019-11-14; most recent amendment 2026-04-24.
Status change: Active, Not Recruiting → Recruiting 2022-07-26
Status change: Recruiting → Active, Not Recruiting 2021-04-07
Status change: Not Yet Recruiting → Recruiting 2020-06-22
Trial Details
NCT Number NCT04165486
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Multiple System Atrophy
Enrollment 40 participants
Start Date 2022-07-21
Primary Completion 2027-09 (estimated)
Study Completion 2027-09 (estimated)
Updated on ClinicalTrials.gov 2026-04-27