A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related… (NCT06512454) | Clinical Trial Compass
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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 range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. 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.
Time to Death (All-causes) and Cause-specific Death (Liver Disease-specific Causes)