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Clinical Trials in the UK / NCT04165486
Recruiting Phase 1

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
No
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)

FacilityCityRegionStatus
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

More Ionis Pharmaceuticals, Inc. trials in the UK

An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM) Enrolling by invitation
FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS) Active, not recruiting
HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome Active, not recruiting
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) Active, not recruiting
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) Active, not recruiting

Other trials for the same condition

A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA) Active, not 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