Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
Trial ID or NCT#
Status
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
- * 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
- * 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)
Contact us to find out if this trial is right for you.
Contact
Swati Toppo
650-497-4151
View on ClinicalTrials.gov