Alpha-1 Antitrypsin Deficiency Clinical Trial
Official title:
QUANTitative Chest Computed Tomography UnMasking Emphysema Progression in Alpha-1 Antitrypsin Deficiency
Individuals with a deficiency of alpha-1 antitrypsin (AAT) often develop emphysema. Traditional lung function tests may not be the most accurate way to measure the progression of emphysema. This study will compare high resolution computed tomography (CT) scans to spirometry to measure the progression of emphysema.
AAT deficiency is a genetic disorder associated with emphysema. Spirometry, the lung function
test that measures how well the lungs exhale air, is used to diagnose and track the
progression of emphysema. Some studies have suggested that forced expiratory volume in 1
second (FEV1) measurements, a type of spirometry test, may lack accuracy in detecting disease
progression in cases of severe AAT deficiency. Another method, high resolution chest CT
scans, may be more accurate at measuring the progression of emphysema. The purpose of this
study is to determine if high resolution CT scans are better at detecting the progression of
emphysema than lung function tests. Results from this study may lead to the development of a
more accurate way to assess lung tissue loss and may improve the understanding of lung
destruction in AAT deficiency.
This study will last 4 years and will enroll people with AAT deficiency who have nearly
normal lung function test results. Study visits, each lasting about 4 hours, will occur at
baseline and months 6, 12, 18, 24, and 36. At each visit, participants will undergo lung
function tests, a CT scan, blood collection, and a physical exam. Female participants will
have urine collected for a pregnancy test. All participants will also complete questionnaires
to assess health status and lung function. Study researchers will call participants every 2
months to collect information on lung disease symptoms and medication changes.
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