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

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.

Timeline

Start

2024-12-17

Primary completion

2026-12

Completion

2026-12

Drugs

Indications