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

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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 Type

Observational


Primary Outcome

CT density slope


Condition

Alpha-1 Antitrypsin Deficiency



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

49

Start Date

August 2007

Completion Date

April 2012

Primary Completion Date

February 2012

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosis of AAT deficiency, as determined by both of the following conditions:

               1. Serum A1-P1 levels less than 11uM or 80 mg/dL

               2. 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

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Charlie Strange, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00532805

Organization ID

RDCRN 5701

Secondary IDs

1U54RR019498-01

Responsible Party

Principal Investigator

Study Sponsor

Medical University of South Carolina

Collaborators

 National Center for Research Resources (NCRR)

Study Sponsor

Charlie Strange, MD, Study Chair, Medical University of South Carolina


Verification Date

January 2018