Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2

Plain-language summary

This study is evaluating an injectable drug called ARO-ATXN2, compared with placebo, in adults with spinocerebellar ataxia type 2 (SCA2). It enrolls adults with a diagnosis of symptomatic SCA2 who have at least 33 CAG repeats in the ATXN2 gene and meet a certain ataxia score threshold. Sponsored by Arrowhead Pharmaceuticals, the study is in Phase 1 and is currently active but not recruiting new participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Active, not recruiting

Sponsor

Arrowhead Pharmaceuticals

Intervention

ARO-ATXN2 Injection, Placebo

Start date

12/17/2024

Estimated completion

12/1/2026

Countries

Australia, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan

Related genes

Locations

• Sydney, Australia
• Melbourne, Australia
• Montreal, Canada
• Montreal, Canada
• Edmonton, Canada
• Paris, France
• Tübingen, Germany
• Ulm, Germany
• Milan, Italy
• Auckland, New Zealand
• Christchurch, New Zealand
• Barcelona, Spain
• Barcelona, Spain
• Seville, Spain
• Kaohsiung City, Taiwan
• Taipei, Taiwan

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.