A Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT


About this study

The purpose of this study 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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Diagnosis of Alpha-1 Antitrypsin Deficiency (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.

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.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Hugo Vargas, M.D.

Open for enrollment

Contact information:

Erica Boyd R.N., CCRP



More information


Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available


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