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

Last updated: March 13, 2025
Sponsor: Arrowhead Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1

Condition

Friedreich's Ataxia

Spinocerebellar Disorders

Dyskinesias

Treatment

Placebo

ARO-ATXN2 Injection

Clinical Study ID

NCT06672445
AROATXN2-1001
2024-514763-25
UTN U111-1308 5875
  • Ages 18-65
  • All Genders

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Non-pregnant, non-lactating

  • Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on sourceverifiable medical records or genetic testing at Screening

  • Scale of Assessment and Rating of Ataxia (SARA) score ≤14

  • Coagulation parameters within normal ranges at Screening

  • Subjects of childbearing potential must agree to use highly effective contraceptionin addition to a condom during the study and for at least 90 days following the endof the study or last dose of study drug, whichever is later. Subjects must notdonate sperm or eggs during the study and for at least 90 days following the end ofthe study or last dose of study drug whichever is later

Exclusion

Exclusion Criteria:

  • Uncontrolled hypertension (blood pressure >160/100 mmHg)

  • History of having received stem cell therapy

  • Clinically significant cardiac, liver, or renal disease

  • Human immunodeficiency virus (HIV) infection (seropositive at Screening)

  • Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening

  • Intellectual disability or significant behavioral neuropsychiatric manifestation

  • Any contraindications to lumbar puncture

  • Presence of an implanted shunt for drainage of CSF or an implanted central nervoussystem (CNS) catheter

Note: Additional inclusion/exclusion criteria may apply per protocol.

Study Design

Total Participants: 36
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1
Study Start date:
December 17, 2024
Estimated Completion Date:
September 30, 2026

Connect with a study center

  • Research Site

    Sydney, New South Wales 2170
    Australia

    Site Not Available

  • Research Site

    Melbourne, Victoria 3004
    Australia

    Active - Recruiting

  • Research Site 1

    Montréal, Quebec H3A 2B4
    Canada

    Active - Recruiting

  • Research Site 2

    Montréal, Quebec H2X 0C1
    Canada

    Active - Recruiting

  • Research Site

    Auckland, 0622
    New Zealand

    Active - Recruiting

  • Research Site

    Christchurch, 8011
    New Zealand

    Active - Recruiting

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