New drug aims to tackle rare brain disease
NCT ID NCT06672445
First seen Jun 26, 2026 · Last updated Jun 30, 2026 · Updated 3 times
Summary
This early-stage trial tests a single injection of ARO-ATXN2 in 36 adults with spinocerebellar ataxia type 2, a rare genetic disorder that affects movement and coordination. The main goal is to see if the drug is safe and how the body processes it. Participants are randomly assigned to receive either the drug or a placebo.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ARO-ATXN2 injection
What this could lead to
If successful, this could point toward a treatment that slows or stops the progression of spinocerebellar ataxia type 2.
What could go wrong
This is a very early Phase 1 trial with only 36 people, focused on safety. It may not show any benefit, and side effects are unknown.
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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Locations
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Research Site 1
Montreal, Quebec, H3A 2B4, Canada
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Research Site 10
Barcelona, 08035, Spain
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Research Site 11
Barcelona, 08036, Spain
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Research Site 12
Seville, 41013, Spain
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Research Site 13
Tübingen, Baden-Wurttemberg, 72026, Germany
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Research Site 14
Ulm, Baden-Wurttemberg, 89081, Germany
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Research Site 15
Paris, 75651, France
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Research Site 16
Milan, 20133, Italy
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Research Site 2
Montreal, Quebec, H2X 0C1, Canada
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Research Site 3
Christchurch, 8011, New Zealand
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Research Site 4
Auckland, 0622, New Zealand
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Research Site 5
Kaohsiung City, 833401, Taiwan
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Research Site 6
Taipei, 112201, Taiwan
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Research Site 7
Melbourne, Victoria, 3004, Australia
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Research Site 8
Sydney, New South Wales, 2170, Australia
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Research Site 9
Edmonton, 8440, Canada