Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
Trial ID or NCT#
NCT03945292
Status
RECRUITING
Purpose
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
Official Title
A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]
Eligibility Criteria
Ages Eligible for Study: 18 Years to 75 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
- - Diagnosis of AATD - Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year - 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
Investigator(s)
Paul Kwo
Hepatologist,
Transplant hepatologist,
Gastroenterologist
Professor of Medicine (Gastroenterology and Hepatology)
Contact us to find out if this trial is right for you.
Contact
Swati Toppo
650-497-4151
View on ClinicalTrials.gov
