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Also known as ARO-AAT Injection, Fazirsiran Injection, TAK-999
ARO-AAT · 2 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| ARO-AAT 100 mg Cohort 1b | EXPERIMENTAL | Primary Study Period (6-12 months): 100 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 100 mg dose of subcutaneous AROAAT every 12 weeks (Q12W). Treatment Extension II (up to 24 Months): 100 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT 200 mg Cohort 1 | EXPERIMENTAL | Primary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 3 doses, with 2 optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT 200 mg Cohort 2 | EXPERIMENTAL | Primary Study Period (6-12 months): 200 mg dose of subcutaneous ARO-AAT for a minimum of 5 doses, with optional treatment extension periods. Treatment Extension I (12 months): 200 mg dose of subcutaneous AROAAT Q12W. Treatment Extension II (up to 24 Months): 200 mg dose of subcutaneous ARO-AAT Q12W. |
| ARO-AAT | ACTIVE_COMPARATOR | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ARO-AAT | DRUG | solution for subcutaneous injection |
| ARO-AAT Injection | DRUG | Single or multiple doses of ARO-AAT by subcutaneous (sc) injections |
| Sterile Normal Saline (0.9% NaCl) | OTHER | Calculated volume to match active comparator |
Inclusion Criteria: * Diagnosis of AATD * Liver biopsy indicating Metavir F1-F3 liver fibrosis based on local pathology read. * Women of childbearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use contraception * Willing to provide written inform...
ARO-AAT is an investigational RNA therapy being developed for alpha 1-antitrypsin deficiency, a rare genetic disorder. It is specifically studied in patients with alpha 1-antitrypsin deficiency associated liver disease. The drug is designed to reduce production of the abnormal alpha 1-antitrypsin protein that accumulates in the liver.
ARO-AAT targets alpha 1-antitrypsin, the protein that is deficient or abnormal in alpha 1-antitrypsin deficiency. By targeting this protein, the RNA therapy aims to reduce its production and accumulation in the liver, addressing the underlying cause of liver disease in affected patients.
ARO-AAT is being developed by Arrowhead Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ARWR. The company is responsible for the clinical development of this RNA therapy for alpha 1-antitrypsin deficiency associated liver disease.
ARO-AAT is in Phase 2 clinical development. It has completed two trials: a Phase 1 study in normal adult volunteers and a Phase 2 study in patients with alpha 1-antitrypsin deficiency associated liver disease. The drug is investigational and has not been approved by the FDA.
ARO-AAT has been studied in two completed clinical trials. The Phase 2 trial, NCT03946449, enrolled 16 patients with alpha 1-antitrypsin deficiency associated liver disease in Austria, Germany, and the United Kingdom. The Phase 1 trial, NCT03362242, enrolled 45 healthy adult volunteers in New Zealand.
Yes, ARO-AAT Injection is another name for ARO-AAT. Both refer to the same investigational RNA therapy developed by Arrowhead Pharmaceuticals for alpha 1-antitrypsin deficiency associated liver disease.