Actively Recruiting
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-04-27
40
Participants Needed
15
Research Sites
266 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Screening DaTscan showing loss of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism
- Diagnosed with probable or possible multiple system atrophy (MSA), parkinsonian-type or cerebellar-type
- Able to walk unassisted for at least 10 meters (approximately 30 feet)
You will not qualify if you...
- Presence of cognitive dysfunction with Montreal Cognitive Assessment (MoCA) score less than 25
- Family history of ataxia or parkinsonism with a known genetic cause of these conditions
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 15 locations
1
Medizinische Universität Innsbruck
Innsbruck, Austria, 6020
Actively Recruiting
2
CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL
Lyon, France, 69500
Actively Recruiting
3
Groupe Hospitalier Pitie-Salpetriere
Paris, France, 75013
Actively Recruiting
4
Hopital Purpan
Toulouse, France, 31059
Actively Recruiting
5
Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, Germany, 89081
Actively Recruiting
6
Universitaetsklinikum Duesseldorf AoeR
Düsseldorf, Germany, 40225
Actively Recruiting
7
University Medical Center Göttingen, Clinic for Neurology
Göttingen, Germany, 37075
Actively Recruiting
8
Medizinische Hochschule Hannover (MHH)
Hanover, Germany, 30625
Completed
9
University Hospital Marburg
Marburg, Germany, 35043
Actively Recruiting
10
Klinikum der Universtiatet Muenchen -Campus Grosshadern
München, Germany, 81377
Actively Recruiting
11
Hospital Beatriz Ângelo
Loures, Portugal, 2674-514
Actively Recruiting
12
Institute of Neurology & The National Hospital for Neurology and Neurosurgery
London, England, United Kingdom, WC1N 3BG
Actively Recruiting
13
The John Radcliffe Hospital
Oxford, England, United Kingdom, OX3 9DU
Actively Recruiting
14
Salford Royal Hospital
Salford, United Kingdom, M6 8HD
Actively Recruiting
15
Southampton General Hospital
Southampton, United Kingdom, SO16 6YD
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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