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New drug aims to tackle rare brain disease

NCT ID NCT06672445

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

39 people

The number who actually took part.

Started

Dec 2024

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Non-pregnant, non-lactating * Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on source verifiable medical records or genetic testing at Screening * Scale of Assessment and Rating of Ataxia (SARA) score ≤14 * Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later Exclusion Criteria: * Uncontrolled hypertension (blood pressure \>160/100 mmHg) * History of having received stem cell therapy * Clinically significant cardiac, liver, or renal disease * Human immunodeficiency virus (HIV) infection (seropositive at Screening) * Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening * Intellectual disability or significant behavioral neuropsychiatric manifestation * Any contraindications to lumbar puncture, including INR \>1.4, platelet count \<100,000, and use of anticoagulant or antiplatelet medications that cannot be safely interrupted * Presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter Note: Additional inclusion/exclusion criteria may apply per protocol.

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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

More trials for these conditions

Other studies related to the condition(s) this trial covers.