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RECRUITING
NCT06512454

A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems

Sponsor: Takeda

View on ClinicalTrials.gov

Summary

The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of AAT, called Z-AAT. Z-AAT builds up in liver cells and also leads to low blood levels of AAT (called Alpha-1 Antitrypsin Deficiency or AATD). Over time, this build up leads to different stages of liver problems, if not treated. This is called natural history of AATD. The main aim of this study is to learn about liver problems caused by AATD in adults when not treated over 4 to 8 years. Other aims are to learn what can predict the AATD-liver condition starting and getting better or worse, describe how this condition is currently being diagnosed and watched in normal care, and describe how the AATD also affects an adult's lung function. Data in this study will be collected to include medical history of a participant, including the date AATD was first identified and/or the date on which the first AATD-related liver or lung problems were diagnosed. At study start and then every year until study end, participants will be asked to complete questionnaires (called patient-reported outcomes or PROs).

Official title: Prospective Observational Study on the Natural History of Alpha-1 Antitrypsin Deficiency and Associated Liver Disease

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

OBSERVATIONAL

Enrollment

500

Start Date

2024-09-25

Completion Date

2031-12-31

Last Updated

2026-07-24

Healthy Volunteers

No

Interventions

OTHER

No Intervention

This is an observational study.

Locations (8)

University of Florida

Gainesville, Florida, United States

University of South Carolina

Charleston, South Carolina, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Vienna General Hospital (AKH Wien)

Vienna, Austria

Universitätsklinikum Aachen

Aachen, Germany

Beaumont Hospital

Dublin, Ireland

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom