No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
18 and older
Sex
Any
Study type
Observational
Participants needed
80 (estimated)
Sponsor
Columbia University · Other
Who this trial is looking for
This trial is looking for adults with a specific genetic condition called PiMZ alpha-1 antitrypsin deficiency who have breathing problems. Participants will undergo lung scans and provide health information to help understand their condition better.
Are You a Good Fit for This Trial?
Rules you outCurrent lung malignancyCOPD exacerbationMajor lung surgeryDecompensated cirrhosis
You may be able to join if
I am at least 18 years old
I understand what the trial involves
I have been diagnosed with PiMZ alpha-1 antitrypsin deficiency
I have reduced lung function as measured by a lung test
I am a member of the Alpha-1 Foundation Clinical Cohort
I agree to share my data with the Alpha-1 Foundation Research Registry
You may not be able to join if
I do not have PiMZ alpha-1 antitrypsin deficiency
I have had a significant lung cancer or other organ cancer in the past 3 years
I have had a lung infection in the past 6 weeks
I am currently pregnant
I cannot lie still for a lung scan
I have allergies to albuterol
I have had major lung surgery in the past
I have a serious lung condition that requires treatment
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
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 …
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.
Trial Locations
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Status
Eligibility Criteria
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…
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
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