Alpha-1 Antitrypsin Deficiency Adult Clinical and Genetic Linkage Study

Status: Active_not_recruiting
Location: See all (3) locations...
Intervention Type: Procedure
Study Type: Observational
SUMMARY

The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed. In addition, the investigators believe that the genetic and environmental factors that play an important role in the development of alpha-1 antitrypsin (AAT) liver disease, can be identified by comparing a cohort database of clinical disease information to linked biospecimen and DNA samples.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• Adults (≥ 18 years of age), with Alpha-1 Antitrypsin Deficiency

• Documented evidence Pi-ZZ phenotype or genotype

• Both genders, all races and ethnic groups

• Willingness to be followed for up to 5 years

• Adults (≥ 18 years of age), with alpha-1-antitrypsin deficiency

• Documented evidence PI-ZZ phenotype or genotype

• Documented evidence of portal hypertension or evidence of advanced liver disease defined by Child-Pugh Class B or C (score ≥ 7), or previous liver biopsy with an Ishak Fibrosis Score ≥ 4

• Both genders, all races and ethnic groups

• Willingness to be followed for up to 5 years

• Adults (≥ 18 years of age), with alpha-1-antitrypsin deficiency

• Pre-transplant documented evidence of PI-ZZ phenotype or genotype

• Documented evidence of liver transplantation

• Both genders, all races and ethnic groups

Locations
United States
California
University of California
San Diego
Massachusetts
Boston University School of Medicine
Boston
Missouri
Saint Louis University
Saint Louis
Time Frame
Start Date: 2013-12
Completion Date: 2026-07
Participants
Target number of participants: 120
Treatments
Liver Biopsy
Participants will provide liver tissue specimens collected at the time of liver biopsy, to determine the rate of progression of liver injury in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency.
Known Severe Liver Disease
Participants will provide samples of serum, plasma, and DNA at defined time points to determine what genetic and environmental modifiers and biomarkers are associated with severe clinical liver disease, such cirrhosis, portal hypertension and liver failure in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency.
Post Liver Transplant
Participants will provide a DNA sample to determine what genetic and environmental modifiers are associated with the need for liver transplantation in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency.
Sponsors
Leads: St. Louis University
Collaborators: University of California, Alpha-1 Foundation, Boston University, University College, London, University of Massachusetts, Worcester

This content was sourced from clinicaltrials.gov