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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 main aim of this study is to learn about liver problems caused by the lack of alpha-1 antitrypsin (called Alpha-1 Antitrypsin Deficiency or AATD) in adults when not treated (this is called the natural history of a condition) over 5 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 hospital care, and describe how the AATD also affects and 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 completed questionnaires (called patient-reported outcomes or PRO).

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

2032-04-06

Last Updated

2025-10-24

Healthy Volunteers

No

Interventions

OTHER

No Intervention

This is a non-interventional study.

Locations (2)

University of Florida

Gainesville, Florida, United States

Universitätsklinikum Aachen AöR

Aachen, North Rhine-Westphalia, Germany