Phase
Condition
Friedreich's Ataxia
Spinocerebellar Disorders
Dyskinesias
Treatment
Placebo
ARO-ATXN2 Injection
Clinical Study ID
Ages 18-65 All Genders
Study Summary
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
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, including INR >1.4, platelet count <100,000, and use of anticoagulant or antiplatelet medications that cannot be safelyinterrupted
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
Connect with a study center
Research Site
Sydney, New South Wales 2170
AustraliaSite Not Available
Research Site
Sydney 2147714, New South Wales 2155400 2170
AustraliaActive - Recruiting
Research Site
Melbourne, Victoria 3004
AustraliaSite Not Available
Research Site
Melbourne 2158177, Victoria 2145234 3004
AustraliaActive - Recruiting
Research Site 1
Montréal, Quebec H3A 2B4
CanadaSite Not Available
Research Site 2
Montréal, Quebec H2X 0C1
CanadaActive - Recruiting
Research Site 1
Montreal 6077243, Quebec 6115047 H3A 2B4
CanadaActive - Recruiting
Research Site 2
Montreal 6077243, Quebec 6115047 H2X 0C1
CanadaActive - Recruiting
Research Site
Auckland, 0622
New ZealandSite Not Available
Research Site
Auckland 2193733, 0622
New ZealandActive - Recruiting
Research Site
Christchurch, 8011
New ZealandSite Not Available
Research Site
Christchurch 2192362, 8011
New ZealandActive - Recruiting
Research Site 1
Kaohsiung, 833401
TaiwanSite Not Available
Research Site 1
Kaohsiung City 1673820, 833401
TaiwanActive - Recruiting
Research Site 2
Taipei, 112201
TaiwanSite Not Available
Research Site 2
Taipei 1668341, 112201
TaiwanActive - Recruiting
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