ARO-ATXN2 Injection: single dose of ARO-ATXN2 by intrathecal (IT) administration
Placebo: calculated volume to match active treatment by IT administration
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
Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria:
* Non-pregnant, non-lactating
* 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:
* 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 safely interrupted
* Presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter
Note: Additional inclusion/exclusion criteria may apply per protocol.
Primary outcome measure(s)
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time — Through End of Study (EOS), Day 253
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.