A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related… (NCT06512454) | Clinical Trial Compass
RecruitingNot Applicable
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
United States, Germany500 participantsStarted 2024-09-25
Plain-language 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).
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. 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).
✓. Cohort 2 (AATD-Pi\*SZ genotype/phenotype with liver disease manifestation).
Exclusion criteria
✕. 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.
✕
What they're measuring
1
Number of Participants With Liver Disease Progression
Timeframe: Baseline up to 5 years
2
Time to Liver Disease Progression
Timeframe: Baseline up to 5 years
3
Time to Liver Disease Trajectory
Timeframe: Baseline up to 5 years
4
Probability of Transition in Liver Disease Trajectory
Timeframe: Baseline up to 5 years
5
Percentage of Participants With Disease Regression
Timeframe: Baseline up to 5 years
6
Time to Liver Disease Regression
Timeframe: Baseline up to 5 years
7
Percentage of Participants With All-cause Mortality and Cause-specific Mortality
Timeframe: Baseline up to 5 years
8
Time to Death (All-causes) and Cause-specific Death (Liver Disease-specific Causes)