PiMZ Longitudinal Cohort (PiMZ Logic)

ConditionsAlpha 1-Antitrypsin Deficiency Emphysema or COPD

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age18+

SponsorColumbia University

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial population

Patients with Alpha-1 Antitrypsin Deficiency genotype MZ across the United States.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

80 Participants

are grouped into 1 trial group

Group A: Emphysema

Trial outcomes

Primary outcomes

Change in lung density over three years

Change in lung density over three years determined by using the 15th percentile point (PERC-15) of Hounsfield units in inspiratory high resolution CT scans.

Time frame

3 years

Decline of PERC-15 (15th percentile) over three years

Rate of decline of PERC-15 over three years. PERC-15 provides the Hounsfield unit point below which 15% of all voxels are distributed. The lowest threshold is -1,000 HU. The lower the PERC-15 values are, the more CT-quantified emphysema is present.

Time frame

3 years

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Columbia University

Lead sponsor

Alpha-1 Foundation

Collaborator

Source ClinicalTrials.gov

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