Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

• Status: Completed
• Trial end date: 2020-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The aim of this study is to test whether aspirin improves endothelial function in alpha-1 antitrypsin deficiency-associated lung disease, measured by pulmonary microvascular blood flow on magnetic resonance imaging (MRI) and with apoptotic endothelial microparticles.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Columbia University
• Collaborators: Alpha-1 Foundation; Stony Wold-Herbert Fund, Inc.
• Treatments: Alpha 1-Antitrypsin; Aspirin; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Alpha-1 antitrypsin deficiency (PiZZ genotype)

• 40 years of age or older

• Evidence of emphysema on CT scan as read by a Radiologist

Exclusion Criteria:

• Platelet count < 150,000/dL, history of intracranial hemorrhage or severe GI bleed, use of systemic anticoagulant, physician prescribed use of antiplatelet drug (including aspirin and P2Y12 receptor inhibitors), or known severe liver disease

• Immunosuppression by use of medications (including oral prednisone), or those with immunomodulatory disease (organ transplantation, autoimmune conditions or actively-treated malignancy)

• Known atrial fibrillation or left ventricular (LV) systolic heart failure

• Contraindication to MRI, including pregnancy, weight > 300 lbs (due to weight limits of the machine), those with pacemakers, aneurysm clips, cochlear implants or other implanted electronic devices, or severe claustrophobia;

• Chronic renal insufficiency (estimated GFR < 45 L/min/1.73 m2 or self report) due to slightly increased risk of nephrogenic systemic fibrosis from gadolinium administration and aspirin-related renal insufficiency

• Exacerbation of respiratory symptoms within the previous 6 weeks, such as that requiring hospitalization, oral prednisone or antibiotics to control symptoms.