Actively Recruiting
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Led by Takeda · Updated on 2025-10-24
500
Participants Needed
2
Research Sites
392 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing to provide written informed consent or currently enrolled in an ongoing participating AATD patient registry that does not require reconsenting
- 18 years of age or older at enrollment
- Documented diagnosis of AATD meeting one of the following:
- Pi*ZZ genotype/phenotype confirmed by genetic assay, sequencing, PCR, or iso-electric focusing electrophoresis
- Pi*SZ genotype/phenotype confirmed by genetic assay, sequencing, PCR, or iso-electric focusing electrophoresis with moderate-advanced or severe liver disease shown by biopsy or laboratory/imaging tests
You will not qualify if you...
- Documented AATD genotype/phenotype other than PiZZ or PiSZ
- History of liver transplant
- No biopsy, magnetic resonance elastography (MRE), fibro scan (VCTE), or APRI results within 24 months before enrollment and none ordered during the enrollment period
- Previous treatment or active participation in an interventional trial for liver or lung disease
- Treatment with liver-directed AATD investigational therapy as part of compassionate use
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
University of Florida
Gainesville, Florida, United States, 32611
Actively Recruiting
2
Universitätsklinikum Aachen AöR
Aachen, North Rhine-Westphalia, Germany, 52074
Actively Recruiting
Research Team
T
Takeda Contact
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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