Injection treatment for 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
This study is testing if an injection called ARO-ATXN2 can help adults with Spinocerebellar Ataxia Type 2 who have a certain genetic marker feel better and improve their condition.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 36 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Locations | 16 sites (Sydney, New South Wales and 15 other locations) |
| Trial ID | NCT06672445 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and effectiveness of ARO-ATXN2 injection in adults diagnosed with Spinocerebellar Ataxia Type 2 who have a specific genetic marker. Participants will be randomly assigned to receive either the ARO-ATXN2 treatment or a placebo. The study will assess various parameters including safety, tolerability, and how the drug is processed in the body. Eligible participants must meet specific genetic and health criteria to ensure accurate results.
Who should consider this trial
Good fit: Ideal candidates are adults with symptomatic Spinocerebellar Ataxia Type 2 carrying at least 33 CAG repeats in the ATXN2 gene.
Not a fit: Patients with uncontrolled hypertension or significant comorbidities such as cardiac, liver, or renal disease may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for managing Spinocerebellar Ataxia Type 2.
How similar studies have performed: While this approach is novel for Spinocerebellar Ataxia Type 2, similar gene-targeted therapies have shown promise in other neurological conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Sydney, New South Wales and 15 other locations
- Research Site 8 — Sydney, New South Wales, Australia (Recruiting)
- Research Site 7 — Melbourne, Victoria, Australia (Recruiting)
- Research Site 2 — Montreal, Quebec, Canada (Recruiting)
- Research Site 1 — Montreal, Quebec, Canada (Recruiting)
- Research Site 9 — Edmonton, Canada (Recruiting)
- Research Site 15 — Paris, Paris, France (Recruiting)
- Research Site 13 — Tübingen, Baden-Wurttemberg, Germany (Recruiting)
- Research Site 14 — Ulm, Baden-Wurttemberg, Germany (Recruiting)
- Research Site 16 — Milan, Italy (Recruiting)
- Research Site 4 — Auckland, New Zealand (Recruiting)
- Research Site 3 — Christchurch, New Zealand (Recruiting)
- Research Site 10 — Barcelona, Spain (Recruiting)
- Research Site 11 — Barcelona, Spain (Recruiting)
- Research Site 12 — Seville, Spain (Recruiting)
- Research Site 5 — Kaohsiung City, Taiwan (Recruiting)
- Research Site 6 — Taipei, Taiwan (Recruiting)
Study contacts
- Study coordinator: Medical Monitor
- Email: AROATXN2@arrowheadpharma.com
- Phone: 626-304-3400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.