HomeTrialNCT04165486
🔬NCT04165486Phase 1RECRUITING

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

Study 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).

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment40 participants
Primary Completion
Lead SponsorIonis Pharmaceuticals, Inc.
Data Retrieved2026-04-11 04:17:53.139385+00:00
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