Not Yet RecruitingNot Applicable

Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.

45 participantsStarted 2026-10-01

Plain-language summary

* To define the course of AATD-associated liver disease. * To use the obtained samples for biomedical research which includes: 1. Search for serum-based disease biomarkers and the associated molecular pathways. 2. Multi-omic spatial analysis of human AATD-LD.

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: * Adult patients (≥18 years) with genetically confirmed alpha-1 antitrypsin deficiency (Pi\*ZZ genotype). * Availability of longitudinal clinical follow-up data (minimum 5 years) within the AATD consortium. * At least one documented liver assessment including liver stiffness measurement (LSM) and serum-based fibrosis markers. * Availability of stored serum samples for proteomic analysis. * For translational analyses: availability of liver tissue samples (pediatric or adult) and/or induced pluripotent stem cell (iPSC)-derived hepatocyte-like cells. Exclusion Criteria: * Presence of other chronic liver diseases (e.g., viral hepatitis, autoimmune hepatitis) that may confound fibrosis assessment. * History of liver transplantation prior to study inclusion. * Incomplete clinical, laboratory, or follow-up data. * Poor-quality or insufficient biological samples for proteomic or molecular analyses. * Patients lost to follow-up or with unreliable longitudinal data

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

Search for serum-based disease biomarkers and the associated molecular pathways

Timeframe: Eight months of performing serum proteomics to identify biomarkers that reflect liver disease severity and predict poor outcomes

Trial details

NCT IDNCT07639996

SponsorAssiut University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-09-30

View on ClinicalTrials.gov More Alpha 1-antitrypsin Deficiency (AATD) trials

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.