PiMZ Longitudinal Cohort: tracking lung changes in PiMZ Alpha-1 patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

Researchers will follow adults with confirmed PiMZ alpha-1 antitrypsin deficiency and obstructive lung disease, collecting high-resolution chest CTs, blood and sputum biomarkers, spirometry, and questionnaires at enrollment, 18 months, and 36 months. CT lung density will be measured to see if baseline emphysema predicts subsequent change in emphysema or biomarker levels, with monthly exacerbation questionnaires between visits. Participants will be existing members of the Alpha-1 Foundation Research Registry and procedures include induced sputum at some sites, blood draws, and supine CT imaging. The overarching goal is to identify early markers that can be used to select PiMZ patients for future interventional trials because traditional lung function measures may not capture progression.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Males and females aged 18 years and older
2. Understand the study procedures, risks, benefits, purpose
3. Able and willing to comply with the study procedures
4. Have PiMZ alpha-1 antitrypsin deficiency
5. Post bronchodilator FEV1 \< 80% predicted AND post bronchodilator FEV1/FVC \< 70%
6. Be an existing member of the Alpha-1 Foundation Clinical Cohort (also known as the Alpha-1 Foundation Research Registry)
7. Agree to have the data collected in this study be shared with the Alpha-1 Foundation Research Registry

Exclusion Criteria:

1. AATD non-PiMZ status, including carriers
2. Current lung, hematologic, or solid organ malignancy other than skin or cervical Stage 1 cancers within the past 3 years
3. COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit
4. Pregnancy at the time of the screening visit
5. Inability to lie still in a supine position for 15 minutes during CT acquisition
6. Inability to perform quality-controlled lung function testing
7. Allergy to albuterol
8. Currently receiving intravenous or subcutaneous immunoglobulin for any disease state
9. Past or present major surgery on the lungs including pneumonectomy or lobectomy. Wedge resections, past segmentectomy, and pleurodesis surgeries are allowed.
10. Previous lung or liver transplantation or currently on the transplant list
11. Decompensated cirrhosis
12. Current presence of endobronchial coils or valves in the lung
13. 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.
14. 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.
15. Patient with Automatic Implantable Cardioverter Defibrillator (AICD) and permanent pacemakers (PPM)
16. Patient receiving biologic immunomodulators that will affect the assessment of the serum biomarkers (as determined by the site PI)
17. Patient with pleural catheters
18. Any condition that in the opinion of the investigator might adversely influence the study outcome

Where it's running

• University of California- Los Angeles
  Los Angeles, California, United States Recruiting
• University of Alabama at Birmingham
  Birmingham, Alabama, United States Not yet recruiting
• National Jewish Health
  Denver, Colorado, United States Not yet recruiting
• University of Chicago
  Chicago, Illinois, United States Not yet recruiting
• Columbia University Irving Medical Center
  New York, New York, United States Not yet recruiting

Study contacts

• Principal investigator: Monica Goldklang, MD — Columbia University
• Study coordinator: Sabrina Palumbo, BS
• Email: sp4461@cumc.columbia.edu
• Phone: 2123053745

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.