Loading clinical trials...

Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2 | Clinical Trials | Clareo Health

RECRUITINGPHASE1

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

A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2

NCT06672445•Arrowhead Pharmaceuticals

View on ClinicalTrials.gov

Summary

Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.

Eligibility

Age

18 - 65 years

Sex

ALL

Healthy Volunteers

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

Locations (16)

Research Site 8

Sydney, New South Wales, Australia

Research Site 7

Melbourne, Victoria, Australia

Research Site 2

Montreal, Quebec, Canada

Research Site 1

Montreal, Quebec, Canada

Research Site 9

Edmonton, Canada

Research Site 15

Paris, Paris, France

Research Site 13

Tübingen, Baden-Wurttemberg, Germany

Research Site 14

Ulm, Baden-Wurttemberg, Germany

Research Site 16

Milan, Italy

Research Site 4

Auckland, New Zealand

Key Dates

Start Date

December 17, 2024

Primary Completion Date

December 1, 2026

Completion Date

December 1, 2026

Last Updated

February 6, 2026

Enrollment

ESTIMATED participants

Conditions

Spinocerebellar Ataxia Type 2

Interventions

ARO-ATXN2 Injection

DRUG

Placebo

DRUG

Contacts

Medical Monitor

CONTACT

626-304-3400 AROATXN2@arrowheadpharma.com

Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)

NCT03347344

Spinocerebellar Ataxia Type 2

View study

WITHDRAWNPHASE2

Study of CAD-1883 for Spinocerebellar Ataxia

NCT04301284

Spinocerebellar AtaxiasSpinocerebellar Ataxia Type 1+8 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 6, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions