Status:

COMPLETED

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

Lead Sponsor:

University of Florida

Conditions:

Liver Fibrosis

Alpha-1 Antitrypsin Deficiency

Eligibility:

All Genders

18-70 years

Brief 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 in...

Detailed Description

Our overarching hypothesis is that liver disease in adults with AAT deficiency is the result of the accumulation of the abnormally folded protein within the endoplasmic reticulum of the hepatocyte. In...

Eligibility Criteria

Inclusion

  • 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

  • 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.

Key Trial Info

Start Date :

October 1 2013

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

September 1 2019

Estimated Enrollment :

109 Patients enrolled

Trial Details

Trial ID

NCT01810458

Start Date

October 1 2013

End Date

September 1 2019

Last Update

August 21 2023

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Shands at the University of Florida

Gainesville, Florida, United States, 32610

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