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

NCT04165486 · tracked via the Priya Life Science UK tracker

Sponsor

Ionis Pharmaceuticals, Inc.

Phase

Phase 1

Started

2022-07-21

Last updated

2026-04-27

Condition(s) studied

Multiple System Atrophy

Investigational drug(s) / intervention(s)

ION464Placebo

ION464: ION464 will be administered by IT injection.

Placebo: ION464-matching placebo will be administered by IT injection.

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).

Eligibility

Sex

ALL

Min age

40 Years

Max age

70 Years

Healthy volunteers

Key Inclusion Criteria: * Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read. * Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C). * Must be able to walk unassisted for at least 10 meters (approximately 30 feet) Key Exclusion Criteria: * Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score \<25) * Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism. NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

Primary outcome measure(s)

Number of Participants with Adverse Events (AEs) — Baseline up to approximately 36 weeks
Number of Participants with Serious Adverse Events (SAEs) — Baseline up to approximately 36 weeks

Trial sites (15)

Facility	City	Region	Status
Medizinische Universität Innsbruck	Innsbruck	Austria	Recruiting
CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL	Lyon	France	Recruiting
Groupe Hospitalier Pitie-Salpetriere	Paris	France	Recruiting
Hopital Purpan	Toulouse	France	Recruiting
Universitaetsklinikum Ulm	Ulm	Baden-Wurttemberg	Recruiting
Universitaetsklinikum Duesseldorf AoeR	Düsseldorf	Germany	Recruiting
University Medical Center Göttingen, Clinic for Neurology	Göttingen	Germany	Recruiting
Medizinische Hochschule Hannover (MHH)	Hanover	Germany	Completed
University Hospital Marburg	Marburg	Germany	Recruiting
Klinikum der Universtiatet Muenchen -Campus Grosshadern	München	Germany	Recruiting
Hospital Beatriz Ângelo	Loures	Portugal	Recruiting
Institute of Neurology & The National Hospital for Neurology and Neurosurgery	London	England	Recruiting
The John Radcliffe Hospital	Oxford	England	Recruiting
Salford Royal Hospital	Salford	United Kingdom	Recruiting
Southampton General Hospital	Southampton	United Kingdom	Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04165486 on ClinicalTrials.gov ↗ ← All trials in the UK