Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03008915
• Date of registration: 22/12/2016
• Prospective Registration: Yes
• Primary sponsor: Columbia University
• Public title: Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
• Scientific title: Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
• Date of first enrolment: January 2017
• Target sample size: 15
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03008915
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Other. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Carrie Aaron, MD
• Affiliation: Columbia University

Key inclusion & exclusion 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.



Age minimum: 40 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Emphysema

Alpha-1 Antitrypsin Deficiency

Chronic Obstructive Pulmonary Disease

Intervention(s)

Drug: Aspirin

Drug: Placebo

Other: Withdrawal from alpha1 antitrypsin replacement therapy

Primary Outcome(s)

Pulmonary microvascular blood flow [Time Frame: 2 weeks]

Secondary Outcome(s)

Pulmonary microvascular blood flow [Time Frame: 5 weeks]

Endothelial apoptosis [Time Frame: 2 weeks]

Endothelial apoptosis [Time Frame: 5 weeks]

Secondary ID(s)

AAAP9855

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Alpha-1 Foundation

Stony Wold-Herbert Fund, Inc.