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

NCT00532805 | Completed DMC

• 2 other identifiers: RDCRN 57011U54RR019498-01
• Study Type: observational
• Target: 49
• Locations: 1 country
• Sites: 7

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.

Conditions

Alpha-1 Antitrypsin Deficiency

Keywords

Alpha 1-Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (1)

• CT density slope

  3 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Medical University of South Carolina: lead
• National Center for Research Resources (NCRR): collaborator
• Alpha-1 Foundation: collaborator

Study Sites (7)

National Jewish Medical and Research Center

Denver, Colorado, United States

Location

University of Florida Medical Center

Gainesville, Florida, United States

Location

Harvard/Brigham and Women's Hospital

Boston, Massachusetts, United States

Location

Cincinnati Children's Medical Center

Cincinnati, Ohio, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Location

Oregon Health and Sciences University

Portland, Oregon, United States

Location

Medical University of South Carolina

Charleston, South Carolina, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

50 cc of serum at visits baseline, 6 months, 12 months, 18 months, 24 months and 36 months will be retained. These are kept at the University of Florida in the laboratory of Dr. Mark Brantly. There is an associated but independent DNA collection that is done if the patient is willing through an independent study and consent process with the University of Florida Alpha-1 DNA and Tissue Bank. This is a public resource with a scientific advisory committee with samples available for researcher access.

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Charlie Strange, MD

  Medical University of South Carolina

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pulmonary Medicine

Study Record Dates

• First Submitted: September 18, 2007
• First Posted: September 20, 2007
• Study Start: August 1, 2007
• Primary Completion: February 1, 2012
• Study Completion: April 1, 2012
• Last Updated: January 23, 2018

Record last verified: 2018-01

Locations

US (7)