Actively Recruiting

Age: 18Years +
All Genders
ID06505603

Alpha-1 Antitrypsin PiMZ Longitudinal Cohort Study to Track Lung Disease Progression

Led by Columbia University · Updated on 2026-01-16

80

Participants Needed

5

Research Sites

N/A

Total Duration

On this page

Sponsors

C

Columbia University

Lead Sponsor

A

Alpha-1 Foundation

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying lung disease progression in people with Alpha-1 Anti-trypsin Deficiency (AATD), focusing on those with the PiMZ genotype. This genetic condition can cause lung and liver problems, and its effects vary among individuals. The goal is to find early signs in lung scans and blood or sputum tests that predict worsening lung disease in these patients, as traditional lung function tests may not always reflect disease progression accurately. The study follows a group of PiMZ patients with obstructive lung disease over three years. Participants will undergo detailed lung imaging using high-resolution CT scans at the start and again at 18 and 36 months. Blood and sputum samples will be collected to measure various biomarkers. Participants also complete questionnaires about their lung health, including monthly surveys about exacerbations. These procedures help researchers track changes in lung density and other markers over time. Participants will have their medical and medication histories reviewed and perform lung function tests such as spirometry at each visit. The study will assess changes in lung density and lung function decline, using CT imaging and biomarker data to identify early signs of worsening emphysema. The total study duration for each participant is three years, with regular follow-ups to monitor lung health and disease progression.

CONDITIONS

Brief Title

PiMZ Longitudinal Cohort (PiMZ Logic)

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Males and females aged 18 years and older
  • Understand and agree to study procedures, risks, benefits, and purpose
  • Able and willing to follow study procedures
  • Have confirmed PiMZ alpha-1 antitrypsin deficiency
  • Post bronchodilator FEV1 less than 80% predicted and FEV1/FVC ratio less than 70%
  • Current member of the Alpha-1 Foundation Clinical Cohort or Research Registry
  • Agree to share data with the Alpha-1 Foundation Research Registry
Not Eligible

You will not qualify if you...

  • Non-PiMZ alpha-1 antitrypsin deficiency status, including carriers
  • Current lung, blood, or solid organ cancer except certain early-stage skin or cervical cancers within 3 years
  • Lung infection or COPD exacerbation within 6 weeks before the first visit
  • Pregnancy at screening
  • Unable to lie still in a supine position for 15 minutes during CT scan
  • Unable to perform quality lung function tests
  • Allergy to albuterol
  • Receiving intravenous or subcutaneous immunoglobulin treatment
  • Major past or present lung surgery (except some minor surgeries)
  • Previous or planned lung or liver transplant
  • Decompensated liver cirrhosis
  • Presence of lung coils or valves
  • Significant bronchiectasis with recent infections
  • Active participation in other therapeutic trials (except certain alpha-1 therapies)
  • Having an implantable cardioverter defibrillator or permanent pacemaker
  • Receiving biologic immunomodulators affecting biomarkers
  • Having pleural catheters
  • Any condition that may interfere with study outcomes

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Diagnostic Evaluation

Duration - 1 day

Participants undergo baseline assessments including medical history review, blood draw, spirometry, induced sputum (at some sites), completion of questionnaires, and a high resolution CT chest scan.

1 visit (in-person)

Long-term Monitoring

Duration - 3 years

Participants are followed longitudinally to assess lung disease progression with repeated assessments at 18 months and 36 months including blood draw, spirometry, induced sputum (at some sites), questionnaires, and high resolution CT chest scans. Additionally, participants complete monthly Alpha-net exacerbation questionnaires.

Assessments at enrollment, 18 months, and 36 months; monthly questionnaires

Trial Site Locations

Total: 5 locations

1

University of Alabama at Birmingham

Birmingham, Alabama, United States, 35233

Not Yet Recruiting

2

University of California- Los Angeles

Los Angeles, California, United States, 90095

Actively Recruiting

3

National Jewish Health

Denver, Colorado, United States, 80206

Not Yet Recruiting

4

University of Chicago

Chicago, Illinois, United States, 60637

Not Yet Recruiting

5

Columbia University Irving Medical Center

New York, New York, United States, 10032

Not Yet Recruiting

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Research Team

S

Sabrina Palumbo, BS

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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