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

• Register: ClinicalTrials.gov
• Last refreshed on: 28 August 2023
• Main ID: NCT01810458
• Date of registration: 06/03/2013
• Prospective Registration: Yes
• Primary sponsor: University of Florida
• Public title: Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)
• Scientific title: Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin Deficiency
• Date of first enrolment: October 2013
• Target sample size: 109
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT01810458
• Study type: Observational

Countries of recruitment

United States

Contacts

• Name: Mark Brantly, MD
• Affiliation: University of Florida

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

AAT Deficiency

AATD

Alpha-1 Antitrypsin Deficiency

Liver Fibrosis

Intervention(s)

Device: Abdominal ultrasound

Drug: Acetaminophen

Drug: Fentanyl

Drug: Lidocaine

Other: Liver questionnaire

Procedure: Blood draw

Drug: Ondansetron

Drug: Midazolam

Procedure: Liver Biopsy

Procedure: History and physical

Procedure: Intravenous catheter

Drug: Lorazepam

Drug: Oxycodone/Acetaminophen

Primary Outcome(s)

To estimate the prevalence and histologic spectrum of liver injury in an adult with Alpha-1 Antitrypsin deficiency having a ZZ genotype or other rare allele. [Time Frame: up to 30 days]

Secondary Outcome(s)

To define the diagnostic accuracy of non-invasive markers of fibrosis in AAT liver disease. [Time Frame: At the screening and year 3 visits.]

To explore epigenetic markers for the development of liver fibrosis. [Time Frame: Starting with the first liver biopsy and ending with the second liver biopsy done at year 3.]

To identify environmental and host risk factors for clinically significant liver fibrosis. [Time Frame: At each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits.]

To quantify liver fibrosis progression. [Time Frame: At each study visit including screening, first liver biopsy, year 1, year 2, and year 3 visits.]

Secondary ID(s)

910

IRB201601019

IRB201601019/ 63-2013

Source(s) of Monetary Support

Please refer to primary and secondary sponsors