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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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Research Site 1

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • Research Site 10

    RECRUITING

    Barcelona, 08035, Spain

  • Research Site 11

    RECRUITING

    Barcelona, 08036, Spain

  • Research Site 12

    RECRUITING

    Seville, 41013, Spain

  • Research Site 13

    RECRUITING

    Tübingen, Baden-Wurttemberg, 72026, Germany

  • Research Site 14

    RECRUITING

    Ulm, Baden-Wurttemberg, 89081, Germany

  • Research Site 15

    RECRUITING

    Paris, Paris, 75651, France

  • Research Site 16

    RECRUITING

    Milan, 20133, Italy

  • Research Site 2

    RECRUITING

    Montreal, Quebec, H2X 0C1, Canada

  • Research Site 3

    RECRUITING

    Christchurch, 8011, New Zealand

  • Research Site 4

    RECRUITING

    Auckland, 0622, New Zealand

  • Research Site 5

    RECRUITING

    Kaohsiung City, 833401, Taiwan

  • Research Site 6

    RECRUITING

    Taipei, 112201, Taiwan

  • Research Site 7

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • Research Site 8

    RECRUITING

    Sydney, New South Wales, 2170, Australia

  • Research Site 9

    RECRUITING

    Edmonton, 8440, Canada

Conditions

Explore the condition pages connected to this study.