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Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

NCT ID: NCT05297812

Last Updated: 2024-12-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

286 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-03-23

Study Completion Date

2027-01-31

Brief Summary

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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.

Detailed Description

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Progression of lung disease in patients with Alpha-1 Anti-trypsin deficiency is variable and while some patients may have stable lung function over many years, some progress and deteriorate rapidly. Currently there are no predictors that would help identify patient at risk of rapid deterioration. The aim of this study is to identify markers and features in CT imaging that may allow identification of these patients early before deterioration. To achieve this, the study will follow a cohort of patients with confirmed Alpha-1 Anti-trypsin deficiency and lung disease and measure a number of biomarkers in blood and sputum and obtain high resolution CT scans at baseline and again three years later. If the study is able to determine markers that allow the identification of patients at risk early, the investigator may be able to study early interventions in later studies and possibly find ways to avoid serious complications. Patients will be followed longitudinally to assess deterioration of lung function.

Study procedures include: Review of medical history and medication history, blood draw, complete Pulmonary Function Test (PFT), induced sputum (at some sites), completion of questionnaires and CT Chest scan. All of the mentioned procedures above will be performed on enrollment and repeated at 18 months and 36 months, with the exception of monthly Alpha- net exacerbation questionnaires.

Conditions

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Alpha 1-Antitrypsin Deficiency Genetic Disease

Keywords

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Alpha-1 Biomarker PiZZ

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Moderate to Advanced Emphysema

Patients with moderate or advanced Emphysema as measured by baseline inspiratory PERC-15 below the study median

No interventions assigned to this group

Minimal Emphysema

Patients with minimal emphysema as measured by baseline inspiratory PERC-15 above the study median

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. Males and females aged 18 years and older
2. Patients with known Alpha-1 Anti-trypsin deficiency (PiZZ)
3. Be an existing member of the Alpha-1 Foundation Clinical Cohort (Alpha-1 Foundation Research Registry)
4. Willingness to perform study procedures of Chest CT, blood biomarker determination, questionnaire completion, and lung function testing.

Exclusion Criteria

1. AATD non-PiZZ status, including carriers
2. Pregnancy at the time of the screening visit
3. COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit
4. Previous lung or liver transplantation or currently on the transplant list
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alpha-1 Foundation

OTHER

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Columbia University

OTHER

Sponsor Role lead

Responsible Party

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Jeanine D'Armiento

Associate Professor of Medicine (in Anesthesiology)

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Jeanine M D'Armiento, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Charlie Strange, MD

Role: STUDY_CHAIR

Medical University of South Carolina

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

University of California - Los Angeles

Los Angeles, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

Boston University

Boston, Massachusetts, United States

Site Status

Columbia University

New York, New York, United States

Site Status

University of North Carolina

Chapel Hill, North Carolina, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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1UG3HL152323

Identifier Type: NIH

Identifier Source: secondary_id

View Link

AAAS8713

Identifier Type: -

Identifier Source: org_study_id