Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Research type
Research Study
Full title
A Phase 1 Placebo-Controlled Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects with Spinocerebellar Ataxia Type 2
IRAS ID
1011358
Contact name
Regulatory Affairs Department
Contact email
Sponsor organisation
Arrowhead Pharmaceuticals, Inc.
Eudract number
2024-514763-25
Research summary
Spinocerebellar ataxias (SCAs) are a group of inherited neurological disorders (disorders that affect the brain and nerves) that are characterised by coordination problems that get worse over time. These disorders can lead to severe disability and premature death. SCA Type 2 (SCA2) is a common form of SCA. Some symptoms of SCA2 include difficulty with balance, speaking and swallowing, confusion or memory loss, and slow eye movements. Arrowhead Pharmaceuticals, Inc., has developed a potential treatment called ARO-ATXN2 that may lower the amount of ATXN2 protein in the body. Lowering this protein may stop or slow the disease progression in participants with SCA2.
The purposes of this study are to:
• Evaluate how safe and well-tolerated ARO-ATXN2 is.
• Evaluate how much of the dose and how quickly ARO-ATXN2 is absorbed into the blood and cerebrospinal fluid (CSF) and then broken down or eliminated from the body (pharmacokinetics (PK)).
• Measure the body’s response to ARO-ATXN2.
ARO-ATXN2 has not been tested in humans before. It is not known what side effects might occur, and it is not known whether ARO-ATXN2 will have any effect on SCA2.
ARO-ATXN2 is an experimental treatment. This means that it is not an approved treatment for SCA2 in the United States or other countries.REC name
South Central - Oxford C Research Ethics Committee
REC reference
25/SC/0090
Date of REC Opinion
5 Jun 2025
REC opinion
Further Information Favourable Opinion