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

RecruitingPhase 1Ages 40 Years - 70 YearsAllIonis Pharmaceuticals, Inc.
NCT04165486 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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).

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📋 Eligibility rules (as published)
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.
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What it tests

  • ION464
  • Placebo
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Where

15 sites
Medizinische Universität Innsbruck
Innsbruck, Austria
CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL
Lyon, France
Groupe Hospitalier Pitie-Salpetriere
Paris, France
Hopital Purpan
Toulouse, France
Universitaetsklinikum Ulm
Ulm, Germany
Universitaetsklinikum Duesseldorf AoeR
Düsseldorf, Germany
University Medical Center Göttingen, Clinic for Neurology
Göttingen, Germany
Medizinische Hochschule Hannover (MHH)
Hanover, Germany

…and 7 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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