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Clinical Trials in Canada / NCT06672445
Active, not recruiting Phase 1

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

NCT06672445 · tracked via the Priya Life Science Canada tracker
Phase
Phase 1
Started
2024-12-17
Last updated
2026-06-29

Condition(s) studied

Spinocerebellar Ataxia Type 2

Investigational drug(s) / intervention(s)

ARO-ATXN2 InjectionPlacebo

ARO-ATXN2 Injection: single dose of ARO-ATXN2 by intrathecal (IT) administration

Placebo: calculated volume to match active treatment by IT administration

Study 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

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (16)

FacilityCityRegionStatus
Research Site 8 Sydney New South Wales
Research Site 7 Melbourne Victoria
Research Site 2 Montreal Quebec
Research Site 1 Montreal Quebec
Research Site 9 Edmonton Canada
Research Site 15 Paris France
Research Site 13 Tübingen Baden-Wurttemberg
Research Site 14 Ulm Baden-Wurttemberg
Research Site 16 Milan Italy
Research Site 4 Auckland New Zealand
Research Site 3 Christchurch New Zealand
Research Site 10 Barcelona Spain
Research Site 11 Barcelona Spain
Research Site 12 Seville Spain
Research Site 5 Kaohsiung City Taiwan
Research Site 6 Taipei Taiwan

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06672445 on ClinicalTrials.gov ↗ ← All trials in Canada