Observational Study of Liver Problems in Alpha-1 Antitrypsin Deficiency (AATD)
This study aims to understand how liver problems develop and change over time in adults with Alpha-1 Antitrypsin Deficiency (AATD). AATD is a condition where the liver makes an abnormal protein (Z-AAT) that builds up in liver cells, leading to liver issues. This is an observational study, meaning you won't receive any new treatments; researchers will simply follow your health over 4 to 8 years. They want to learn what factors predict liver disease getting better or worse, how it's currently diagnosed and managed, and how AATD also affects the lungs. The study is looking for 500 adults, aged 18 and older, who have a confirmed diagnosis of AATD (specifically the Pi*ZZ genotype/phenotype). The study's success will be measured by tracking how liver disease progresses, how long it takes for changes to occur, and the overall path of liver disease over this 8-year period. The current recruitment status is unclear.
- Study design
- This is an observational study, meaning no intervention is given. It plans to enroll 500 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 8 years to track liver disease progression.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
At a glance
Conditions
Where it's being run
8 sites across 8 statesStudy leadership
- Study Director · STUDY_DIRECTOR · Takeda
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Inclusion
Exclusion
What this trial measures
- Number of Participants With Liver Disease ProgressionBaseline up to 8 years
Liver disease progression will be defined as advancement in greater than or equal to (\>=)1 fibrosis stage: example any progression from fibrosis stage F0/F1 to F2, F1 to F2, F2 to F3 etc. and/or occurrence of any of these composite events: a) advancement in \>=1 fibrosis stage, b) development of a liver disease-related clinical event, c) model for end-stage liver disease (MELD) score increase, d) newly added on liver transplant list, e) receipt of a liver transplant. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis. The MELD score ranges from 6 to 40 with higher scores indicating more severe liver disease and a worse outcome.
- Time to Liver Disease ProgressionBaseline up to 8 years
Time to liver disease progression is defined as time to advancement in \>=1 fibrosis stage (example F0/F1 to F2, F2 to F3 etc.) and/or time to the earliest of: Advancement in \>=1 fibrosis stage, or development of a liver disease-related clinical event, or MELD score increase or receipt/newly added to transplant list of a liver transplant. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis. The MELD score ranges from 6 to 40 with higher scores indicating more severe liver disease and a worse outcome.
- Time to Liver Disease TrajectoryBaseline up to 8 years
Time to liver disease trajectory is defined as time of transition from F0/F1 to F2, F2 to F3, F3 to F4, F4 to the first decompensating event or liver transplant/listing and first to second decompensating event and all subsequent decompensating events or liver transplant/listing. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis.
- Probability of Transition in Liver Disease TrajectoryBaseline up to 8 years
Probability of liver disease trajectory is defined as probability of transition from F0/F1 to F2, F2 to F3, F3 to F4, F4 to the first decompensating event or liver transplant/listing and first to second decompensating event and all subsequent decompensating events or liver transplant/listing. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis.
- Percentage of Participants With Disease RegressionBaseline up to 8 years
Disease regression is defined as decrease in \>=1 fibrosis staging. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis.
- Time to Liver Disease RegressionBaseline up to 8 years
Time to liver disease regression is defined as time to decrease in \>=1 fibrosis stage. The fibrosis stages range from F0 to F4, with F0 indicating no fibrosis and F4 indicating cirrhosis.
- Percentage of Participants With All-cause Mortality and Cause-specific MortalityBaseline up to 8 years
Cause-specific mortality is defined as mortality due to liver failure or complications of cirrhosis/portal hypertension or hepatocellular carcinoma, or infections secondary to liver failure.
- Time to Death (All-causes) and Cause-specific Death (Liver Disease-specific Causes)Baseline up to 8 years
Cause-specific mortality is defined as mortality due to liver failure or complications of cirrhosis/portal hypertension or hepatocellular carcinoma, or infections secondary to liver failure.