Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels

• Status: Withdrawn
• Trial end date: 2016-11-01
• Target enrollment: 0
• Participant gender: All

Summary

Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.

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

Details

• Lead Sponsor: Arrowhead Pharmaceuticals
• Treatments: Alpha 1-Antitrypsin; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Male or non-nursing female patients 18-75 years of age, inclusive, at the time of Screening

• Previous diagnosis of PiZZ genotype Alpha-1 Antitrypsin Deficiency (AATD)

• Non-smoker (not a daily cigarette smoker) for at least three years with current non-smoking status confirmed by urine cotinine at screening.

• Highly effective, double barrier contraception (both male and female partners) during the study and for 3 months following the last dose of ARC-AAT Injection

• Suitable venous access for blood sampling

Exclusion Criteria:

• Known diagnosis of hepatic fibrosis from a cause other than AATD

• History of poorly controlled autoimmune disease, or any history of autoimmune hepatitis

• Human immunodeficiency virus (HIV) infection

• Seropositive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV)

• Uncontrolled hypertension

• History of cardiac rhythm disturbances

• Symptomatic heart failure, unstable angina, myocardial infarction, severe cardiovascular disease, transient ischemic attack (TIA) or cerebrovascular accident (CVA) within 6 months prior to study entry

• History of malignancy within the last 2 years except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer

• History of major surgery within 1 month of Screening

• Regular use of alcohol within one month prior to the Screening visit

• Use of illicit drugs (such as cocaine, phencyclidine [PCP] and crack) within 1 year prior to Screening or positive urine drug screen at Screening

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

• Any clinically significant history/presence of an uncontrolled systemic disease

• Blood donation (≥500 mL) within 7 days prior to study treatment administration