New hope for rare brain disease: first human trial launches
NCT ID NCT04165486
First seen Jan 02, 2026 · Last updated May 05, 2026 · Updated 22 times
Summary
This early-stage study tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and serious brain disorder. The main goal is to check the drug's safety and how well people tolerate it when given via a spinal injection. Researchers will also measure changes in a protein linked to the disease to see if the drug is hitting its target.
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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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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
Conditions
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