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Experimental spinal injection aims to slow rare brain disease

NCT ID NCT04165486

Recruiting now Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. The drug is given via a spinal injection and aims to reduce levels of a protein linked to the disease. The main goal is to check safety and tolerability, not yet to prove it works.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
ION464 (an experimental drug given via spinal injection)
What this could lead to
If successful, this could point toward a treatment that slows or stops the progression of multiple system atrophy, a rare and devastating brain disease.
What could go wrong
This is a very early Phase 1 trial with only 40 participants, focused on safety. The drug may not work, could have side effects, and it will take years to know if it truly helps patients.

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Conditions

The condition(s) this trial relates to.

multiple system atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MULTIPLE SYSTEM ATROPHY

Contacts and locations

Locations

  • CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL

    RECRUITING

    Lyon, 69500, France

  • Groupe Hospitalier Pitie-Salpetriere

    RECRUITING

    Paris, 75013, France

  • Hopital Purpan

    RECRUITING

    Toulouse, 31059, France

  • Hospital Beatriz Ângelo

    RECRUITING

    Loures, 2674-514, Portugal

  • Institute of Neurology & The National Hospital for Neurology and Neurosurgery

    RECRUITING

    London, England, WC1N 3BG, United Kingdom

  • Klinikum der Universtiatet Muenchen -Campus Grosshadern

    RECRUITING

    München, 81377, Germany

  • Medizinische Hochschule Hannover (MHH)

    COMPLETED

    Hanover, 30625, Germany

  • Medizinische Universität Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

  • Salford Royal Hospital

    RECRUITING

    Salford, M6 8HD, United Kingdom

  • Southampton General Hospital

    RECRUITING

    Southampton, SO16 6YD, United Kingdom

  • The John Radcliffe Hospital

    RECRUITING

    Oxford, England, OX3 9DU, United Kingdom

  • Universitaetsklinikum Duesseldorf AoeR

    RECRUITING

    Düsseldorf, 40225, Germany

  • Universitaetsklinikum Ulm

    RECRUITING

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University Hospital Marburg

    RECRUITING

    Marburg, 35043, Germany

  • University Medical Center Göttingen, Clinic for Neurology

    RECRUITING

    Göttingen, 37075, Germany

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