RecruitingNCT06512454

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

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


Sponsor

Takeda

Enrollment

500 participants

Start Date

Sep 25, 2024

Study Type

OBSERVATIONAL

Conditions

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).


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying No Intervention for people with alpha1-antitrypsin deficiency. The study is currently recruiting participants at 8 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERNo 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

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NCT06512454


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