Protocol No.: OCR21902
Sponsor Protocol No.: AROAAT2001
Protocol Title.: Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT
Principal Investigator: Clark, Virginia
Objective: The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, tolerability and effect on liver histologic parameters with administration of the investigational product, ARO-AAT, in participants with alpha-1 antitrypsin deficiency (AATD). Participants will receive multiple subcutaneous doses of ARO-AAT.
Description: In Part A, participants will be enrolled to receive multiple doses of ARO-AAT or placebo at varying dose levels. Once Part A is complete, a single dose level for Part B will be selected based on safety and pharmacodynamic parameters from Part A. Patients enrolled into Part A will roll over to the Part B dose level or continue to receive placebo.
Phase: Phase II
Age Group: Adult
Age: 18 Years - 75 Years
Experimental: ARO-AATPart A: administered on Days 1, 29 and 113 and every 84 days thereafter until dose selected for Part B Part B: minimum of 6, maximum of 9 doses
Placebo Comparator: PlaceboPart A: administered on Days 1, 29 and 113 and every 84 days thereafter until dose selected for Part B Part B: minimum of 6, maximum of 9 doses
Detailed Eligibility: Inclusion Criteria: - Diagnosis of AATD - Liver biopsy at Screening indicating liver fibrosis - 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