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Study identifier: NCT04165486 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Multiple System Atrophy  ·  Sponsor: Ionis Pharmaceuticals, Inc.

PhasePhase 1
Planned participants40
Who can joinAll sexes, 40 Years to 70 Years
Healthy volunteersNo

About this study

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).

This description comes directly from the study's public registry record.

Talk to the study team

Ionis Pharmaceuticals, Inc.  ·  (844) 748-5844  ·  IonisHORIZONStudy@clinicaltrialmedia.com

Always discuss trial participation with your own doctor first.

Locations (15)

Medizinische Universität InnsbruckInnsbruck, AustriaRecruiting
CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCLLyon, FranceRecruiting
Groupe Hospitalier Pitie-SalpetriereParis, FranceRecruiting
Hopital PurpanToulouse, FranceRecruiting
Universitaetsklinikum UlmUlm, Baden-Wurttemberg, GermanyRecruiting
Universitaetsklinikum Duesseldorf AoeRDüsseldorf, GermanyRecruiting
University Medical Center Göttingen, Clinic for NeurologyGöttingen, GermanyRecruiting
Medizinische Hochschule Hannover (MHH)Hanover, GermanyCompleted
University Hospital MarburgMarburg, GermanyRecruiting
Klinikum der Universtiatet Muenchen -Campus GrosshadernMünchen, GermanyRecruiting
Hospital Beatriz ÂngeloLoures, PortugalRecruiting
Institute of Neurology & The National Hospital for Neurology and NeurosurgeryLondon, England, United KingdomRecruiting

+ 3 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT04165486