Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

• Status: Active, not recruiting
• Trial end date: 2023-08-23
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the the safety and efficacy of ARO-AAT Injection (also referred to as ARO-AAT) administered subcutaneously to patients with alpha-1 antitrypsin deficiency.

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

Details

• Lead Sponsor: Arrowhead Pharmaceuticals

Criteria

Inclusion Criteria:

• Diagnosis of AATD

• 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 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

• 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