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