New drug aimed at rare brain disease enters first human tests
NCT ID NCT06672445
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 27 times
Summary
This study is testing a new medicine called ARO-ATXN2 for people with spinocerebellar ataxia type 2, a rare genetic brain disorder that affects movement and coordination. The main goal is to check if the drug is safe and how the body processes it. About 36 adults will get either a single dose of the drug or a placebo, and researchers will monitor them for side effects and drug levels in the blood.
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the original study
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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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Research Site 1
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
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Research Site 10
RECRUITINGBarcelona, 08035, Spain
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Research Site 11
RECRUITINGBarcelona, 08036, Spain
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Research Site 12
RECRUITINGSeville, 41013, Spain
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Research Site 13
RECRUITINGTübingen, Baden-Wurttemberg, 72026, Germany
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Research Site 14
RECRUITINGUlm, Baden-Wurttemberg, 89081, Germany
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Research Site 15
RECRUITINGParis, Paris, 75651, France
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Research Site 16
RECRUITINGMilan, 20133, Italy
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Research Site 2
RECRUITINGMontreal, Quebec, H2X 0C1, Canada
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Research Site 3
RECRUITINGChristchurch, 8011, New Zealand
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Research Site 4
RECRUITINGAuckland, 0622, New Zealand
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Research Site 5
RECRUITINGKaohsiung City, 833401, Taiwan
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Research Site 6
RECRUITINGTaipei, 112201, Taiwan
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Research Site 7
RECRUITINGMelbourne, Victoria, 3004, Australia
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Research Site 8
RECRUITINGSydney, New South Wales, 2170, Australia
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Research Site 9
RECRUITINGEdmonton, 8440, Canada
Conditions
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