Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
NCT ID: NCT06672445
Last Updated: 2025-12-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
36 participants
INTERVENTIONAL
2024-12-17
2026-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ARO-ATXN2
ARO-ATXN2 Injection
ARO-ATXN2 Injection
single dose of ARO-ATXN2 by intrathecal (IT) administration
Placebo
(0.9% NaCl)
Placebo
calculated volume to match active treatment by IT administration
Interventions
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ARO-ATXN2 Injection
single dose of ARO-ATXN2 by intrathecal (IT) administration
Placebo
calculated volume to match active treatment by IT administration
Eligibility Criteria
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Inclusion Criteria
* Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on source verifiable medical records or genetic testing at Screening
* Scale of Assessment and Rating of Ataxia (SARA) score ≤14
* Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later
Exclusion Criteria
* 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, including INR \>1.4, platelet count \<100,000, and use of anticoagulant or antiplatelet medications that cannot be safely interrupted
* Presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter
18 Years
65 Years
ALL
No
Sponsors
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Arrowhead Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Research Site 8
Sydney, New South Wales, Australia
Research Site 7
Melbourne, Victoria, Australia
Research Site 2
Montreal, Quebec, Canada
Research Site 1
Montreal, Quebec, Canada
Research Site 9
Edmonton, , Canada
Research Site 4
Auckland, , New Zealand
Research Site 3
Christchurch, , New Zealand
Research Site 10
Barcelona, , Spain
Research Site 11
Barcelona, , Spain
Research Site 12
Seville, , Spain
Research Site 5
Kaohsiung City, , Taiwan
Research Site 6
Taipei, , Taiwan
Countries
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Central Contacts
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Other Identifiers
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UTN U111-1308 5875
Identifier Type: OTHER
Identifier Source: secondary_id
2024-514763-25
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AROATXN2-1001
Identifier Type: -
Identifier Source: org_study_id