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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 designed to target the ATXN2 gene, which is implicated in the disease. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
ARO-ATXN2 targets the ATXN2 gene, which is associated with Spinocerebellar Ataxia Type 2. By targeting this molecular target, the drug aims to address the underlying genetic cause of the condition. This mechanism is being evaluated in early-stage clinical trials to assess its safety and potential efficacy.
ARO-ATXN2 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ARWR. The company is conducting clinical research on this investigational drug for Spinocerebellar Ataxia Type 2, with the drug currently in Phase 1 trials.
ARO-ATXN2 is in Phase 1 clinical development. It is an investigational drug being studied for Spinocerebellar Ataxia Type 2, and it has not yet received regulatory approval. 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 trial is titled 'Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2' and is active but not recruiting. The study enrolls 39 participants across 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 as an injection and is being investigated for the treatment of Spinocerebellar Ataxia Type 2. This alternative name is used in clinical trial documentation to describe the formulation of the investigational therapy.