Phase
Condition
Spinocerebellar Disorders
Friedreich's Ataxia
Dyskinesias
Treatment
ARO-ATXN2 Injection
Placebo
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 8
Sydney, New South Wales 2170
AustraliaSite Not Available
Research Site 7
Melbourne, Victoria 3004
AustraliaSite Not Available
Research Site 1
Montreal, Quebec H3A 2B4
CanadaSite Not Available
Research Site 2
Montreal, Quebec H2X 0C1
CanadaSite Not Available
Research Site 9
Edmonton, 8440
CanadaSite Not Available
Research Site 15
Paris, 75651
FranceSite Not Available
Research Site 13
Tübingen, Baden-Wurttemberg 72026
GermanySite Not Available
Research Site 14
Ulm, Baden-Wurttemberg 89081
GermanySite Not Available
Research Site 16
Milan, 20133
ItalySite Not Available
Research Site 4
Auckland, 0622
New ZealandSite Not Available
Research Site 3
Christchurch, 8011
New ZealandSite Not Available
Research Site 10
Barcelona, 08035
SpainSite Not Available
Research Site 11
Barcelona, 08036
SpainSite Not Available
Research Site 12
Seville, 41013
SpainSite Not Available
Research Site 5
Kaohsiung City, 833401
TaiwanSite Not Available
Research Site 6
Taipei, 112201
TaiwanSite Not Available

Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.