Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT

• Status: Active, not recruiting
• Trial end date: 2022-07-13
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, ARO-AAT, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Arrowhead Pharmaceuticals

Criteria

Inclusion Criteria:

• Diagnosis of AATD

• Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year

• Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception

• Willing to provide written informed consent and to comply with study requirements

• Non-smoker for at least 1 year

• No abnormal finding of clinical relevance at Screening

Exclusion Criteria:

• Clinically significant health concerns other than AATD

• Previous diagnosis or diagnosis at Screening of definitive liver cirrhosis

• Previous lung or liver transplant due to AATD

• Regular use of alcohol within one month prior to Screening

• Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study involving therapeutic intervention

• Use of illicit drugs within 1 year prior to Screening

NOTE: additional inclusion/exclusion criteria may apply, per protocol