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Taking people in United KingdomPhase 1

Testing ION464 given into the spinal fluid for multiple system atrophy

Tests ION464 for safety in adults with multiple system atrophy.

Key facts

Who runs it
Ionis Pharmaceuticals, Inc.
Where
London, Oxford, Salford, and Southampton, United KingdomSee the 4 sitesAlso in 4 other countries, 11 sites
Registry title
Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Status
Recruiting
Phase
Phase 1
People taking part
40 people, planned
Started
21 Jul 2022
Expected to end
Sept 2027
Type of study
Interventional, 4 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • ION464
  • Placebo
Record updated
27 April 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with multiple system atrophy (MSA), a disease that damages the nerves. It tests the safety of ION464, given as an injection into the fluid around the spine.

Age
40 Years to 70 Years
Sex
All sexes can join.
Disease stage
Probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C). People must be able to walk without help for at least 10 meters (approximately 30 feet).
Biomarker or test result
A DaTscan™ brain scan (SPECT) at screening must show loss of dopamine nerve endings in the striatum, a deep part of the brain. This loss must fit with parkinsonism caused by nerve damage.

Main reasons for not taking part

  • Problems with memory or thinking, with a MoCA test score <25
  • A family history of ataxia or parkinsonism together with a known genetic cause of ataxia or parkinsonism
  • Other rules set by the protocol may apply

The registry criteria, word for word

Show all 5 criteriaShow less

Who can take part

  • 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)

Who cannot take part

  • 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)

Check if you might fit

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?

Where in United Kingdom

Also in 4 other countries, 11 sites

  • Institute of Neurology & The National Hospital for Neurology and NeurosurgeryLondon, United KingdomContact: Ionis Pharmaceuticals, Inc.Taking people
  • The John Radcliffe HospitalOxford, United KingdomContact: Ionis Pharmaceuticals, Inc.Taking people
  • Salford Royal HospitalSalford, United KingdomContact: Ionis Pharmaceuticals, Inc.Taking people
  • Southampton General HospitalSouthampton, United KingdomContact: Ionis Pharmaceuticals, Inc.Taking people

About the trial

What the trial wants to find out

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

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).

The 4 groups in the trial

  • Part 1: ION464Treatment being tested

    ION464 will be administered at multiple-ascending doses by IT injection at regular intervals over 12 weeks.

    Treatment: ION464

  • Part 1: PlaceboPlacebo, for comparison

    ION464-matching placebo will be administered by IT injection at regular intervals over 12 weeks.

    Treatment: Placebo

  • Part 2: ION464Treatment being tested

    ION464 will be administered at the same doses as Part 1 by IT injection, at regular intervals, for 72 weeks.

    Treatment: ION464

  • Part 2: PlaceboPlacebo, for comparison

    ION464-matching placebo will be administered by IT injection, at regular intervals, for 72 weeks.

    Treatment: Placebo

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy
Plain-language title
Testing ION464 given into the spinal fluid for multiple system atrophyGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 14 November 2026.
Fit questions
Built from NCT04165486, who can take part
Record updated
27 April 2026