Experimental spinal injection aims to slow rare brain disease
NCT ID NCT04165486
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL
RECRUITINGLyon, 69500, France
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Groupe Hospitalier Pitie-Salpetriere
RECRUITINGParis, 75013, France
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Hopital Purpan
RECRUITINGToulouse, 31059, France
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Hospital Beatriz Ângelo
RECRUITINGLoures, 2674-514, Portugal
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Institute of Neurology & The National Hospital for Neurology and Neurosurgery
RECRUITINGLondon, England, WC1N 3BG, United Kingdom
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Klinikum der Universtiatet Muenchen -Campus Grosshadern
RECRUITINGMünchen, 81377, Germany
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Medizinische Hochschule Hannover (MHH)
COMPLETEDHanover, 30625, Germany
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Medizinische Universität Innsbruck
RECRUITINGInnsbruck, 6020, Austria
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Salford Royal Hospital
RECRUITINGSalford, M6 8HD, United Kingdom
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Southampton General Hospital
RECRUITINGSouthampton, SO16 6YD, United Kingdom
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The John Radcliffe Hospital
RECRUITINGOxford, England, OX3 9DU, United Kingdom
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Universitaetsklinikum Duesseldorf AoeR
RECRUITINGDüsseldorf, 40225, Germany
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Universitaetsklinikum Ulm
RECRUITINGUlm, Baden-Wurttemberg, 89081, Germany
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University Hospital Marburg
RECRUITINGMarburg, 35043, Germany
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University Medical Center Göttingen, Clinic for Neurology
RECRUITINGGöttingen, 37075, Germany