Actively Recruiting
Prospective Observational Study on the Natural History of Alpha-1 Antitrypsin Deficiency and Associated Liver Disease
Led by Takeda · Updated on 2025-10-24
500
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying adults with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can cause liver problems, to understand how these liver issues develop and change over five years without treatment. The study also aims to identify factors that predict when liver disease may begin or improve, describe how the condition is diagnosed and managed in normal hospital care, and assess how AATD affects lung function in adults. Participants diagnosed with specific AATD types—either homozygous Pi*ZZ with mild or no liver disease, or heterozygous Pi*SZ with moderate to severe liver disease—will be followed. Data will be collected during routine care visits over the study period. No treatment is given as part of the study since it is observational, focusing on monitoring and collecting health data. During the study, participants will provide medical history including dates of diagnosis and liver or lung problems. They will complete yearly questionnaires about their health for up to five years. Researchers will track outcomes such as liver disease progression or regression, lung disease development, and mortality causes. The study helps improve understanding of AATD and its effects, with data collected through regular clinical care and patient reports.
CONDITIONS
Brief 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 a participating AATD patient registry without reconsenting
- Age 18 years or older at enrollment
- Documented diagnosis of Alpha-1 Antitrypsin Deficiency (AATD)
- Cohort 1: Pi*ZZ genotype/phenotype confirmed by genetic assay or lab test
- Cohort 2: Pi*SZ genotype/phenotype confirmed by genetic assay or lab test with moderate to severe liver disease confirmed by biopsy or lab/imaging measures
You will not qualify if you...
- AATD genotype/phenotype other than PiZZ or PiSZ
- History of liver transplant
- No recent biopsy, magnetic resonance elastography, fibro scan, or APRI test results within 24 months prior to enrollment and none ordered during enrollment
- Previous treatment or active participation in interventional trials for liver or lung disease
- Treatment with liver-directed AATD investigational therapy as compassionate use
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 5 years
Participants who undergo routine care are observed to learn about the natural history of Alpha-1 Antitrypsin Deficiency and associated liver disease.
Visits occur per routine care during follow-up
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
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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