Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

• Status: Completed
• Trial end date: 2019-09-01
• Target enrollment: 0
• Participant gender: All

Summary

We hypothesize that individuals with Alpha-1 Antitrypsin (AAT) deficiency have ongoing liver injury which is not detected by the usual blood tests used to look at liver function. This ongoing liver injury leads to cirrhosis in a significant number of adults with AAT deficiency.

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Florida
• Treatments: Acetaminophen; Acetaminophen, hydrocodone drug combination; Alpha 1-Antitrypsin; Fentanyl; Lidocaine; Liver Extracts; Lorazepam; Midazolam; Ondansetron; Oxycodone; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Alpha-1 Antitrypsin deficiency confirmed to be PI*ZZ by both genotype or another identified rare allele;

• Age range from 18-70;

• Willingness to consent to liver biopsy;

• Ability to travel to UF as necessary by protocol; and

• Platelet count greater than or equal to 50,000/mm3 and an INR less than or equal to 1.5.

Exclusion Criteria:

• Hemophilia, anticoagulant therapy that cannot be interrupted briefly, malignancy, or any other condition that would compromise the safety of a liver biopsy;

• Any known pre-existing medical condition that might interfere with the patient's participation in and completion of the study or any condition, which in the opinion of the investigator would make the patient unsuitable for enrollment;

• Active substance abuse including, but not limited to, alcohol, intravenous or, inhaled drugs;

• History of adverse reactions or allergy to the local anesthetic, sedative, or pre-medication used for the percutaneous liver biopsy;

• Poor venous access making the subject unable to complete the required laboratory testing schedule; and

• Females who are pregnant or lactating at time of enrollment. Should a female subject become pregnant during the follow up period after the initial liver biopsy, continued participation would be allowed if the following conditions are met: the subject desires to continue; a discussion of risk and benefits of participation between the principal investigator and the subject has occurred; and no liver biopsy would be performed in the follow up period.