Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

Recruiting

I'm Interested

Trial 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
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 Interested

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Swati Toppo
650-497-4151