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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02900183
• Date of registration: 09/09/2016
• Prospective Registration: Yes
• Primary sponsor: Arrowhead Pharmaceuticals
• Public title: Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
• Scientific title: An Open-Label, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Effect on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels of ARC-AAT as Evidenced by Changes in Liver Biopsy in Patients With Alpha-1 Antitrypsin Deficiency (AATD)
• Date of first enrolment: October 2016
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02900183
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Canada, Ireland, Italy, Sweden

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Alpha-1 Antitrypsin Deficiency

Intervention(s)

Drug: ARC-AAT Injection

Primary Outcome(s)

Incidence and frequency of adverse events as a measure of safety and tolerability of multiple doses of ARC-AAT Injection [Time Frame: Baseline through Day 287 End-of Study Visit]

Secondary Outcome(s)

Change from baseline in circulating serum levels of alpha-1 antitrypsin [Time Frame: Baseline through Day 287 End-of Study Visit]

Secondary ID(s)

2016-000917-59

ARCAAT2001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors