NCT06672445

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

A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
DRUG	ARO-ATXN2 Injection	single dose of ARO-ATXN2 by intrathecal (IT) administration
DRUG	Placebo	calculated volume to match active treatment by IT administration

Timeline

• Start date: 2024-12-17
• Primary completion: 2026-12-01
• Completion: 2026-12-01
• First posted: 2024-11-04
• Last updated: 2026-02-06

Locations

16 sites across 8 countries: Australia, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan

Regulatory

• FDA-regulated drug study