Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
A1BC
2 other identifiers
observational
286
1 country
9
Brief Summary
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. The purpose of this study is to examine the density of the lung as measured by chest computed tomography (CT) and determine if existing emphysema predicts changes in the rate of subsequent emphysema or changes in CT, serum or plasma biomarkers of interest. The overarching goal is to develop biomarkers that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Mar 2022
Longer than P75 for all trials
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 17, 2022
CompletedStudy Start
First participant enrolled
March 23, 2022
CompletedFirst Posted
Study publicly available on registry
March 28, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2027
January 16, 2026
January 1, 2026
4.8 years
March 17, 2022
January 14, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in lung density over three years
Change in lung density over three years determined by using the 15th percentile point of Hounsfield units in inspiratory high resolution CT scans (PERC-15)
3 years
Study Arms (2)
Moderate to Advanced Emphysema
Patients with moderate or advanced Emphysema as measured by baseline inspiratory PERC-15 below the study median
Minimal Emphysema
Patients with minimal emphysema as measured by baseline inspiratory PERC-15 above the study median
Eligibility Criteria
Patients with Alpha-1 Antitrypsin Deficiency genotype ZZ across the United States.
You may qualify if:
- Males and females aged 18 years and older
- Patients with known Alpha-1 Anti-trypsin deficiency (PiZZ)
- Be an existing member of the Alpha-1 Foundation Clinical Cohort (Alpha-1 Foundation Research Registry)
- Willingness to perform study procedures of Chest CT, blood biomarker determination, questionnaire completion, and lung function testing.
You may not qualify if:
- AATD non-PiZZ status, including carriers
- Pregnancy at the time of the screening visit
- COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit
- Previous lung or liver transplantation or currently on the transplant list
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Columbia Universitylead
- Alpha-1 Foundationcollaborator
- National Heart, Lung, and Blood Institute (NHLBI)collaborator
Study Sites (9)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
University of California - Los Angeles
Los Angeles, California, 90095, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Chicago
Chicago, Illinois, 60637, United States
Boston University
Boston, Massachusetts, 02118, United States
Columbia University
New York, New York, 10032, United States
University of North Carolina
Chapel Hill, North Carolina, 27514, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
University of Utah
Salt Lake City, Utah, 84108, United States
Biospecimen
Blood samples will be collected for analysis in the ongoing clinical trial as well as for banking to for a bio-repository for future AATD studies. Participants will have the option to agree/not agree to partake in the optional bio-repository.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Jeanine M D'Armiento, MD, PhD
Columbia University
- STUDY CHAIR
Charlie Strange, MD
Medical University of South Carolina
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 36 Months
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Associate Professor of Medicine (in Anesthesiology)
Study Record Dates
First Submitted
March 17, 2022
First Posted
March 28, 2022
Study Start
March 23, 2022
Primary Completion (Estimated)
January 1, 2027
Study Completion (Estimated)
January 1, 2027
Last Updated
January 16, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL
- Time Frame
- After first 3 years of data collection and preliminary analysis
- Access Criteria
- De-identified data will be available via i2b2 with proper protocol and IRB regulatory documentation
This study is part of a larger research study where one of the objectives is to create a AATD de-identified, public use repository of data. This repository will be query based, and any sub-projects that stem from this will need to abide by the appropriate Human Subjects Protection, GCP guidelines (i.e., IRB protocol, local IRB approval, DUA, Release of Information) and be reviewed by the research committee including the Alpha-1 Foundation prior to release of any de-identified data.