Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Key facts
- Who runs it
- Ionis Pharmaceuticals, Inc.
- Where
- Bron, Paris, and Toulouse, FranceSee the 3 sitesAlso in 4 other countries, 12 sites
- Trial code
- NCT04165486
- EU 2024-512528-13-00
- EudraCT 2019-001105-24
- U1111-1308-8964
- 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
- Age
- 40 years to 70 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
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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 France
Also in 4 other countries, 12 sites
- Hospices Civils de LyonBron, FranceTaking people
- Assistance Publique Hopitaux de ParisParis, FranceTaking people
- Centre Hospitalier Universitaire de ToulouseToulouse, FranceTaking 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).
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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
- 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 doctorData 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
- Fit questions
- Built from NCT04165486, who can take part
- Record updated
- 27 April 2026