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Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
Recruiting
I'm InterestedTrial ID: NCT03945292
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]
Stanford Investigator(s)
Paul Kwo
Professor of Medicine (Gastroenterology and Hepatology)
Eligibility
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
Intervention(s):
other: Placebo
drug: Fazisiran Injection
Recruiting
I'm InterestedContact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Swati Toppo
650-497-4151