Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Status:
RECRUITING
Trial end date:
2026-12-01
Target enrollment:
Participant gender:
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.