Clinical Trial

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

Active, Not Recruiting Phase 1
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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.
Protocol Amendment History 1 change
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-06-30
Trial Details
NCT Number NCT06672445
Lead Sponsor Arrowhead Pharmaceuticals
Conditions Spinocerebellar Ataxia Type 2
Enrollment 39 participants
Start Date 2024-12-17
Primary Completion 2026-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2026-06-29