Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Key facts
- Where
- Innsbruck, AustriaSee the site
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 40 and 70 years old?
Source: NCT04165486, who can take part
Where in Austria
- Medizinische Universitaet InnsbruckInnsbruck, AustriaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Ionis Pharmaceuticals, Inc.
- Trial code
- NCT04165486EU 2024-512528-13-00
- Record updated
- 27 April 2026
- Original description
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).Read full original descriptionShow less
A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy
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).
Inclusion: 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).
Exclusion: Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism; NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply; Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score <25).