New drug aims to tackle rare brain disease

NCT ID NCT06672445

Ongoing Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinocerebellar ataxia type 2 Spinocerebellar Ataxias

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINOCEREBELLAR ATAXIA TYPE 2

Contacts and locations

Locations

Research Site 1

Montreal, Quebec, H3A 2B4, Canada
Research Site 10

Barcelona, 08035, Spain
Research Site 11

Barcelona, 08036, Spain
Research Site 12

Seville, 41013, Spain
Research Site 13

Tübingen, Baden-Wurttemberg, 72026, Germany
Research Site 14

Ulm, Baden-Wurttemberg, 89081, Germany
Research Site 15

Paris, 75651, France
Research Site 16

Milan, 20133, Italy
Research Site 2

Montreal, Quebec, H2X 0C1, Canada
Research Site 3

Christchurch, 8011, New Zealand
Research Site 4

Auckland, 0622, New Zealand
Research Site 5

Kaohsiung City, 833401, Taiwan
Research Site 6

Taipei, 112201, Taiwan
Research Site 7

Melbourne, Victoria, 3004, Australia
Research Site 8

Sydney, New South Wales, 2170, Australia
Research Site 9

Edmonton, 8440, Canada

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