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

Launched by ARROWHEAD PHARMACEUTICALS · Sep 9, 2016

Keywords

ClinConnect Summary

This is a multi-center, multi-dose study to evaluate the safety, tolerability and effect on intrahepatic and circulating Alpha-1 Antitrypsin (AAT) levels of ARC-AAT Injection, administered intravenously. Participants who have signed an Institutional Review Board/Ethics Committee (IRB/EC) approved informed consent form and met all of the protocol eligibility criteria will receive multiple intravenous doses (7 total doses administered every 28 days) of ARC-AAT Injection. Patients previously, currently or never receiving AAT augmentation therapy are acceptable for enrollment. All subjects will...

Gender

ALL

Eligibility 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