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

NCT06672445 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2026-02-06

No results posted yet for this 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

DRUG

ARO-ATXN2 Injection

single dose of ARO-ATXN2 by intrathecal (IT) administration

DRUG

Placebo

calculated volume to match active treatment by IT administration

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-17
Primary Completion
2026-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • Australia
  • Canada
  • France
  • Germany
  • Italy
  • New Zealand
  • Spain
  • Taiwan

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06672445 on ClinicalTrials.gov