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

NCT04165486 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2026-04-27

No results posted yet for this 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).

Conditions

Interventions

DRUG

ION464

ION464 will be administered by IT injection.

DRUG

Placebo

ION464-matching placebo will be administered by IT injection.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
SEQUENTIAL

Eligibility

Min Age
40 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-07-21
Primary Completion
2027-09-30
Completion
2027-09-30

Countries

  • Austria
  • France
  • Germany
  • Portugal
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04165486 on ClinicalTrials.gov