The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency

Descriptive Information
Brief Title ^†	The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency
Official Title ^†	QUANTitative Chest Computed Tomography UnMasking Emphysema Progression in Alpha-1 Antitrypsin Deficiency
Brief Summary	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.
Detailed Description	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.
Study Phase	N/A
Study Type ^†	Observational
Study Design ^†	Observational Model: Cohort, Time Perspective: Prospective
Primary Outcome Measure ^†
Secondary Outcome Measure ^†
Condition ^†	Alpha-1 Antitrypsin Deficiency
Intervention ^†
Study Arms / Comparison Groups
Publications *
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status ^†
Estimated Enrollment ^†	60
Start Date ^†	August 2007
Completion Date	August 2011
Primary Completion Date	August 2011
Eligibility Criteria ^†	Inclusion Criteria: Diagnosis of AAT deficiency, as determined by both of the following conditions: Serum A1-P1 levels less than 11uM or 80 mg/dL Protease inhibitor phenotype Z (PiZZ) or Znull phenotype confirmed by gene probe analysis. Previous serum levels and phenotype results are acceptable if documented from a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory. FEV1 greater than or equal to 80% of the predicted value Exclusion Criteria: Pregnant or intending to become pregnant within 4 years of study entry Previous lung transplantation Sibling of a participant who is already enrolled in the study Unable to attend scheduled clinic visits Currently smokes cigarettes or marijuana or quit smoking cigarettes or marijuana in the 1 year prior to study entry Current or planned use of oral tobacco products or nicotine replacement products Evidence of significant long-term or acute inflammation outside the lung, including connective diseases, panniculitis, or acute infection Unwilling to alter bronchodilator medications for 24 hours prior to scheduled quantitative CT (QCT) scans Musculoskeletal disease that limits exercise by walking Required to take any of the following medications within 48 hours of scheduled lung function testing: dicyclomine (Bentyl), propantheline (Pro-Banthine), mepenzolate (Cantil), methscopolamine (Pamine), and scopolamine (Transderm-Scop) Known allergy or intolerance to tiotropium or albuterol
Gender	Both
Ages	18 Years - N/A
Accepts Healthy Volunteers	No
Contacts ^††	, , , ,
Location Countries ^†	United States,
Administrative Information
NCT ID ^†	NCT00532805
Organization ID	RDCRN 5701
Secondary IDs ^††
Responsible Party	Charlie Strange, MD, Medical University of South Carolina
Study Sponsor ^†	Office of Rare Diseases (ORD)
Collaborators ^††
Investigators ^†	Study Chair: Charlie Strange, MD, Medical University of South Carolina
Information Provided By	Office of Rare Diseases (ORD)
Verification Date	July 2009
First Received Date ^†	September 18, 2007
Last Updated Date	July 30, 2009

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.