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

January 21, 2018 updated by: Charlie Strange, Medical University of South Carolina

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.

Study Overview

Status

Completed

Conditions

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

Enrollment (Actual)

49

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Denver, Colorado, United States
        • National Jewish Medical and Research Center
    • Florida
      • Gainesville, Florida, United States
        • University of Florida Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States
        • Harvard/Brigham and Women's Hospital
    • Ohio
      • Cincinnati, Ohio, United States
        • Cincinnati Children's Medical Center
      • Cleveland, Ohio, United States
        • Cleveland Clinic Foundation
    • Oregon
      • Portland, Oregon, United States
        • Oregon Health and Sciences University
    • South Carolina
      • Charleston, South Carolina, United States
        • Medical University of South Carolina

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Individuals with alpha-1 antitrypsin who have nearly normal lung function tests. Participants will be identified from patients of the investigators, physician referral, and the Alpha-1 Foundation Research Registry.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
CT density slope
Time Frame: 3 years
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical University of South Carolina

Collaborators

National Center for Research Resources (NCRR)
Alpha-1 Foundation

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2007

Primary Completion (Actual)

February 1, 2012

Study Completion (Actual)

April 1, 2012

Study Registration Dates

First Submitted

September 18, 2007

First Submitted That Met QC Criteria

September 18, 2007

First Posted (Estimate)

September 20, 2007

Study Record Updates

Last Update Posted (Actual)

January 23, 2018

Last Update Submitted That Met QC Criteria

January 21, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RDCRN 5701
  • 1U54RR019498-01 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Alpha-1 Antitrypsin Deficiency

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Congo, DR of

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe