PiMZ Longitudinal Cohort (PiMZ Logic)
Recruiting · NCT06505603 · Observational (researchers observe without assigning treatment) · Lead sponsor: Columbia University
View the official record on ClinicalTrials.gov →What this trial is about
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. Presentations are variable in the heterozygous population, the most predominant genotype being PiMZ. The purpose of this study in PiMZ heterozygous patients is to examine the density of the lung as measured by chest computed tomography (CT) and determine if existing emphysema predicts changes in the rate of subsequent emphysema or changes in CT, serum or plasma biomarkers of interest. The overarching goal is to develop biomarkers pertinent to the PiMZ patient that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD.
Who can take part
Inclusion criteria
- Males and females aged 18 years and older
- Understand the study procedures, risks, benefits, purpose
- Able and willing to comply with the study procedures
- Have PiMZ alpha-1 antitrypsin deficiency
- Post bronchodilator FEV1 \< 80% predicted AND post bronchodilator FEV1/FVC \< 70%
- Be an existing member of the Alpha-1 Foundation Clinical Cohort (also known as the Alpha-1 Foundation Research Registry)
- Agree to have the data collected in this study be shared with the Alpha-1 Foundation Research Registry
Exclusion criteria
- AATD non-PiMZ status, including carriers
- Current lung, hematologic, or solid organ malignancy other than skin or cervical Stage 1 cancers within the past 3 years
- COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit
- Pregnancy at the time of the screening visit
- Inability to lie still in a supine position for 15 minutes during CT acquisition
- Inability to perform quality-controlled lung function testing
- Allergy to albuterol
- Currently receiving intravenous or subcutaneous immunoglobulin for any disease state
- Past or present major surgery on the lungs including pneumonectomy or lobectomy. Wedge resections, past segmentectomy, and pleurodesis surgeries are allowed.
- Previous lung or liver transplantation or currently on the transplant list
- Decompensated cirrhosis
- Current presence of endobronchial coils or valves in the lung
- Clinically significant bronchiectasis as defined by the investigator. In general, this would exclude patients with chronic infection of the lungs requiring treatment within the past 6 months including non-tuberculous mycobacterial disease, chronic fungal disease, allergic bronchopulmonary aspergillosis, or known colonization of bronchiectasis with pseudomonas or stenotrophomonas species.
- Participation in the active treatment arm of a therapeutic clinical trial at baseline visit unless using one of the Alpha-1 augmentation therapies in alternative doses.
- Patient with Automatic Implantable Cardioverter Defibrillator (AICD) and permanent pacemakers (PPM)
- Patient receiving biologic immunomodulators that will affect the assessment of the serum biomarkers (as determined by the site PI)
- Patient with pleural catheters
- Any condition that in the opinion of the investigator might adversely influence the study outcome
Where it is running
5 locations listed across 5 US states.
- University of Alabama at Birmingham - Birmingham, Alabama, United States
- University of California- Los Angeles - Los Angeles, California, United States
- National Jewish Health - Denver, Colorado, United States
- University of Chicago - Chicago, Illinois, United States
- Columbia University Irving Medical Center - New York, New York, United States