Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2

Launched by ARROWHEAD PHARMACEUTICALS · Nov 1, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ARO-ATXN2 for adults with Spinocerebellar Ataxia Type 2 (SCA2), a condition that affects movement and coordination. In this study, participants who have a specific genetic marker (at least 33 repeats of a certain sequence in the ATXN2 gene) will receive either the new treatment or a placebo (a substance with no active ingredients) in a single injection. The goal is to assess how safe the treatment is, how well it is tolerated, and how it affects the body.

To be eligible for this trial, participants must be adults aged 18 to 74 who have been diagnosed with SCA2 and show certain levels of symptoms. They cannot be pregnant or breastfeeding and should not have serious health issues like uncontrolled high blood pressure or certain infections. During the study, participants will receive close monitoring to ensure their safety. It’s important for those considering joining the trial to understand that they may need to follow specific guidelines, such as using reliable contraception if they could become pregnant. This study is currently recruiting participants, and those interested should discuss it with their healthcare provider to see if they qualify.

Gender

ALL

Eligibility criteria

• 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
• • Coagulation parameters within normal ranges at Screening
• • 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
• • 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.