Hidden liver harm: study tracks silent damage in genetic disorder
NCT ID NCT02014415
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 120 adults with Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause lung and liver problems. Researchers use liver biopsies and genetic samples over 5 years to find out who develops liver scarring or cirrhosis and why. The goal is to identify risk factors that are currently overlooked.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this study could reveal why some people with Alpha-1 Antitrypsin Deficiency develop serious liver disease while others do not, pointing toward future prevention or treatment strategies.
- What could go wrong
- This is an observational study, not a treatment trial, so it will not directly help participants. It is small (120 people) and may not uncover clear genetic or environmental factors.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Boston University School of Medicine
Boston, Massachusetts, 02118, United States
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Saint Louis University
St Louis, Missouri, 63104, United States
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University of California
San Diego, California, 92103, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- AI model could predict who needs a lung transplant for rare emphysema
- Could a simple shot replace IV drips for Alpha-1 patients?
- Promising liver drug trial halted early: what it means for patients
- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition
- Alpha-1 patients donate samples to power future discoveries