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

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.

Conditions

Spinocerebellar Ataxia Type 2

Interventions / Drugs

ARO-ATXN2 Injection Placebo

View on ClinicalTrials.gov ↗

Sponsor

ARWR

Arrowhead Pharmaceuticals

RNAi Therapeutics

D36/100

Study Details

Enrollment36 participants

Primary Completion—

Lead SponsorArrowhead Pharmaceuticals

Data Retrieved2026-04-11 02:54:36.141219+00:00

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