Alpha-1 Antitrypsin DeficiencyQUANTUM-1NCT00532805
The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency
An observational study of Alpha-1 Antitrypsin Deficiency, run by Medical University of South Carolina at 7 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha-1 Antitrypsin Deficiency
- How many people
- 49 participants
- Where
- 7 sitesUnited States
- Study dates
- Aug 2007to Apr 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Harvard/Brigham and Women's Hospital
Boston, Massachusetts
Medical University of South Carolina
Charleston, South Carolina
Cincinnati Children's Medical Center
Cincinnati, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio
National Jewish Medical and Research Center
Denver, Colorado
University of Florida Medical Center
Gainesville, Florida
Oregon Health and Sciences University
Portland, Oregon
About this study
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.
Read the 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.
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Keywords
- Alpha 1-Antitrypsin Deficiency
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What is being measured
The primary outcome is the main question the study is designed to answer.
CT density slope
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2007 (estimated)
- Study startAug 2007
- Primary completionFeb 2012
- Study completionApr 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-1 Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- National Center for Research Resources (NCRR), Alpha-1 Foundation
- Responsible party
- Principal investigator, Charlie Strange, Medical University of South Carolina
- Investigators
- Charlie Strange, MD, Study chair, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00532805
- Sponsor's protocol ID
- RDCRN 5701
- Other IDs
- 1U54RR019498-01
- Acronym
- QUANTUM-1
- Status last verified
- Jan 2018
- First submitted
- 18 Sep 2007
- Last updated
- 23 Jan 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- QUANTitative Chest Computed Tomography UnMasking Emphysema Progression in Alpha-1 Antitrypsin Deficiency
- ClinicalTrials.gov ID
- NCT00532805
- Acronym / sponsor ID
- QUANTUM-1 / RDCRN 5701
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Keywords
- Alpha 1-Antitrypsin Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- National Center for Research Resources (NCRR), Alpha-1 Foundation
- Study start
- Aug 2007
- Primary completion
- Feb 2012 (actual)
- Study completion
- Apr 2012 (actual)
- First posted
- 20 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- Diagnosis of AAT deficiency, as determined by both of the following conditions:
- FEV1 greater than or equal to 80% of the predicted value
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
CT density slope
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Charlie Strange, MD study chair
Medical University of South Carolina
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha-1 Antitrypsin Deficiency, run by Medical University of South Carolina at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
CT density slope (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2018.