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Also known as ARO-ATXN2 Injection
ARO-ATXN2 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| ARO-ATXN2 | EXPERIMENTAL | ARO-ATXN2 Injection |
| Placebo | PLACEBO_COMPARATOR | (0.9% NaCl) |
| Name | Type | Description |
|---|---|---|
| ARO-ATXN2 Injection | DRUG | single dose of ARO-ATXN2 by intrathecal (IT) administration |
| Placebo | DRUG | calculated volume to match active treatment by IT administration |
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 ag...
ARO-ATXN2 is an investigational small molecule being developed for the treatment of Spinocerebellar Ataxia Type 2, a rare inherited neurological disorder. It is administered by injection and is currently in Phase 1 clinical development. The drug is not yet approved by regulatory authorities.
ARO-ATXN2 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARWR. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug for Spinocerebellar Ataxia Type 2.
ARO-ATXN2 is currently in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is active but not recruiting participants, indicating that enrollment has been completed.
ARO-ATXN2 is being studied in a Phase 1 clinical trial with the identifier NCT06672445. This randomized, double-blind, placebo-controlled study enrolls approximately 39 adults with Spinocerebellar Ataxia Type 2. The trial is active but not recruiting and is being conducted in multiple countries including Australia, Canada, France, Germany, Italy, New Zealand, Spain, and Taiwan.
Yes, ARO-ATXN2 is also known as ARO-ATXN2 Injection. The drug is administered via injection, and this alternative name reflects its route of administration. Both names refer to the same investigational drug being developed by Arrowhead Pharmaceuticals for Spinocerebellar Ataxia Type 2.