Status:
RECRUITING
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Lead Sponsor:
Takeda
Conditions:
Alpha1-Antitrypsin Deficiency
Eligibility:
All Genders
18+ years
Brief 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 nat...
Eligibility Criteria
Inclusion
- Participants who meet all the following criteria will be included in the study.
- Cohorts 1 and 2:
- Willing to provide written informed consent or currently enrolled in an ongoing participating AATD patient registry that does not require reconsenting to participate in the study.
- \>=18 years of age at enrollment in this study.
- Participants with documented diagnosis of AATD, meeting the following criteria:
- Cohort 1 (AATD-Pi\*ZZ genotype/phenotype).
- • Pi\*ZZ genotype as documented from rapid genetic assay, sequencing, or polymerase chain reaction (PCR), or Pi\*ZZ phenotype as documented from iso-electric focusing (IEF) electrophoresis.
- Cohort 2 (AATD-Pi\*SZ genotype/phenotype with liver disease manifestation).
- Pi\*SZ genotype as documented from rapid genetic assay, sequencing, or PCR, or Pi\*SZ phenotype as documented from IEF electrophoresis, and
- Moderate-advanced or severe liver disease manifestation as defined by either liver biopsy or surrogate laboratory or imaging measures, as determined through:
- Lab and imaging measures to define liver disease manifestation
Exclusion
- Participants who meet any following criteria will be excluded from the study.
- Documented AATD genotype/phenotype other than Pi\*ZZ or Pi\*SZ.
- History of liver transplant.
- No results for either biopsies, magnetic resonance elastography (MRE), fibro scan (vibration controlled transient elastography \[VCTE\]), or Aspartate aminotransferase to platelet ratio index (APRI) in the 24 months prior to the index/enrollment date and has none of these tests ordered during the index period.
- Participants who had previously been treated or in an active participation in an interventional trial studying liver or lung disease.
- Treatment with liver directed AATD investigational therapy as part of a compassionate use request.
Key Trial Info
Start Date :
September 25 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
April 6 2032
Estimated Enrollment :
500 Patients enrolled
Trial Details
Trial ID
NCT06512454
Start Date
September 25 2024
End Date
April 6 2032
Last Update
October 24 2025
Active Locations (2)
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1
University of Florida
Gainesville, Florida, United States, 32611
2
Universitätsklinikum Aachen AöR
Aachen, North Rhine-Westphalia, Germany, 52074