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PiMZ Longitudinal Cohort (PiMZ Logic)

Alpha-1 Antitrypsin PiMZ Longitudinal Cohort (PiMZ Logic)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Mentioned in the study description

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: CT scans, Blood draws, Questionnaires

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
  • "To achieve this, the study will follow a cohort of patients with confirmed Alpha-1 Anti-trypsin deficiency (MZ genotype) and obstructive lung disease and measure a number of biomarkers in blood and sputum and obtain high resolution CT scans at baseline and again three years later."
  • "Study procedures include: Review of medical history and medication history, blood draw, spriometry, induced sputum (at some sites), completion of questionnaires and CT Chest scan."
  • "All of the mentioned procedures above will be performed on enrollment and repeated at 18 months and 36 months, with the exception of monthly Alpha- net exacerbation questionnaires."
Compensation
Mentioned in the study description
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study follows adults with PiMZ alpha-1 antitrypsin deficiency, an inherited condition that can affect the lungs and liver. It uses chest CT scans and blood tests to see whether existing emphysema, a type of lung damage, predicts future changes. The goal is to find signs of disease changes that can be used in future treatment studies.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You are age 18 or older.
  • You understand the study's purpose, steps, risks, and benefits.
  • You are able and willing to follow the study steps.
  • You have the PiMZ type of alpha-1 antitrypsin deficiency.
  • After medicine to open your airways, your breathing tests show FEV1 below 80% of the expected value and FEV1/FVC below 70%. These tests measure how much air you can blow out.
  • You already belong to the Alpha-1 Foundation Clinical Cohort, also called the Research Registry.
  • You agree to share your study data with the Alpha-1 Foundation Research Registry.

Who can't join

  • You do not have the PiMZ type, including if you carry another type; or you are pregnant at screening.
  • You have current lung, blood, or other organ cancer within the past 3 years, except skin cancer or stage 1 cervical cancer.
  • You had a COPD flare-up or lung infection within 6 weeks before the first study visit; or you have serious bronchiectasis (damaged, widened airways), generally including long-term lung infections needing treatment in the past 6 months or certain bacteria living in the airways.
  • You cannot lie still on your back for a 15-minute CT scan, cannot complete reliable breathing tests, or are allergic to albuterol.
  • You receive immunoglobulin (antibody treatment) through a vein or under the skin, or certain biologic medicines that change the immune system and could affect blood tests. You are in a trial's active treatment group at the first visit, unless it uses an Alpha-1 replacement treatment at a different dose.
  • You have had major lung surgery, such as removal of a lung or lobe. Smaller wedge or segment removals and procedures to stick the lung lining together are allowed. You have had a lung or liver transplant, are on a transplant list, or have severe liver scarring with liver failure.
  • You have coils or valves in your lung, an implanted heart defibrillator or permanent pacemaker, or a tube to drain fluid from around your lung.
  • The study doctor believes another condition could harm the study results.

Simplified for readability. The official criteria below are what the study team uses.

Official 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/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

Study sites (5)

AL

CA

CO

IL

NY