Actively Recruiting
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Led by Arrowhead Pharmaceuticals · Updated on 2026-02-06
36
Participants Needed
16
Research Sites
102 weeks
Total Duration
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AI-Summary
What this Trial Is About
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
Official Title
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Non-pregnant, non-lactating
- Diagnosis of symptomatic SCA2 with 533 CAG repeats in the ATXN2 gene confirmed by medical records or genetic testing
- Scale of Assessment and Rating of Ataxia (SARA) score 14
- Subjects of childbearing potential must agree to use highly effective contraception plus condom during and for at least 90 days after the study or last dose
- Subjects must not donate sperm or eggs during the study and for at least 90 days after the study or last dose
You will not qualify if you...
- Uncontrolled hypertension (blood pressure >160/100 mmHg)
- History of stem cell therapy
- Clinically significant cardiac, liver, or renal disease
- HIV infection (seropositive at screening)
- Seropositive for hepatitis B or C at screening
- Intellectual disability or significant behavioral neuropsychiatric issues
- Contraindications to lumbar puncture including INR >1.4, platelet count <100,000, or use of anticoagulant/antiplatelet drugs that cannot be safely stopped
- Presence of implanted shunt for CSF drainage or implanted central nervous system catheter
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 16 locations
1
Research Site 8
Sydney, New South Wales, Australia, 2170
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2
Research Site 7
Melbourne, Victoria, Australia, 3004
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3
Research Site 2
Montreal, Quebec, Canada, H2X 0C1
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4
Research Site 1
Montreal, Quebec, Canada, H3A 2B4
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5
Research Site 9
Edmonton, Canada, 8440
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6
Research Site 15
Paris, Paris, France, 75651
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7
Research Site 13
Tübingen, Baden-Wurttemberg, Germany, 72026
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8
Research Site 14
Ulm, Baden-Wurttemberg, Germany, 89081
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9
Research Site 16
Milan, Italy, 20133
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10
Research Site 4
Auckland, New Zealand, 0622
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11
Research Site 3
Christchurch, New Zealand, 8011
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12
Research Site 10
Barcelona, Spain, 08035
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13
Research Site 11
Barcelona, Spain, 08036
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14
Research Site 12
Seville, Spain, 41013
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15
Research Site 5
Kaohsiung City, Taiwan, 833401
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16
Research Site 6
Taipei, Taiwan, 112201
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Research Team
M
Medical Monitor
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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