for people ages 18-75 (full criteria)
at Sacramento, California and other locations
study started
estimated completion
Principal Investigator
Brooks Kuhn



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.

Official Title

A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]


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.


Alpha 1-Antitrypsin Deficiency ARO-AAT Injection ARO-AAT


You can join if…

Open to people ages 18-75

  • 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

You CAN'T join if...

  • 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


  • University of California Davis Medical Center accepting new patients
    Sacramento California 95817 United States
  • UCSF Medical Center Benioff Children's Hospital not yet accepting patients
    San Francisco California 94158 United States

Lead Scientist at UC Davis

  • Brooks Kuhn
    Assistant Professor, Pulmonary, Critical Care, and Sleep Medicine. Authored (or co-authored) 15 research publications.


accepting new patients
Start Date
Completion Date
Arrowhead Pharmaceuticals
Sign up for this study
Phase 2/3
Study Type
Last Updated