| NCT ID | NCT06505603 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Columbia University |
| Condition | Alpha 1-Antitrypsin Deficiency |
| Study Type | OBSERVATIONAL |
| Enrollment | 80 participants |
| Start Date | 2025-05-30 |
| Primary Completion | 2029-02 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 80 participants in total. It began in 2025-05-30 with a primary completion date of 2029-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
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
Contact & Investigator
Monica Goldklang, MD
PRINCIPAL INVESTIGATOR
Columbia University
Frequently Asked Questions
Who can join the NCT06505603 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Alpha 1-Antitrypsin Deficiency. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06505603 currently recruiting?
Yes, NCT06505603 is actively recruiting participants. Contact the research team at sp4461@cumc.columbia.edu for enrollment information.
Where is the NCT06505603 trial being conducted?
This trial is being conducted at Birmingham, United States, Los Angeles, United States, Denver, United States, Chicago, United States and 1 additional location.
Who is sponsoring the NCT06505603 clinical trial?
NCT06505603 is sponsored by Columbia University. The principal investigator is Monica Goldklang, MD at Columbia University. The trial plans to enroll 80 participants.