Alpha-1 Antitrypsin Deficiency Clinical Trial
Official title:
The Role of Conformational Diseases on Macrophage Function
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.
Status | Recruiting |
Enrollment | 220 |
Est. completion date | July 20, 2033 |
Est. primary completion date | July 20, 2032 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Signed informed consent - Male or female 18 years of age or older - Negative pregnancy test for women of childbearing potential - Hemoglobin >12.5 g/dl measured on the day of participation - Negative urine nicotine test Exclusion Criteria: - Pregnancy or breastfeeding - Weight < 50 kg - History of anemia requiring blood transfusions, erythropoietin supplementation, or iron supplementation within the past 36 months - Known hemoglobin <12.5 g/dl within the past 90 days - Systolic blood pressure > 180 mmHg and/or diastolic blood pressure >100 mmHg - Poor venous access - Large volume blood donation (>200 ml or 7 ounces) within the previous 56 days (e.g. blood donation for the purposes of blood banking) - Clinically significant cardiac, hemostatic or neurological impairment or any other significant medical condition that, in the opinion of the investigator would affect subject safety (e.g., recent myocardial infarction, history of prolonged bleeding time, cerebral vascular accident, advanced cancer or uncontrolled medical condition) - Psychiatric or cognitive disturbance or illness that would affect subject safety - Current smoker |
Country | Name | City | State |
---|---|---|---|
United States | Shands at the University of Florida | Gainesville | Florida |
Lead Sponsor | Collaborator |
---|---|
University of Florida |
United States,
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Hidvegi T, Schmidt BZ, Hale P, Perlmutter DH. Accumulation of mutant alpha1-antitrypsin Z in the endoplasmic reticulum activates caspases-4 and -12, NFkappaB, and BAP31 but not the unfolded protein response. J Biol Chem. 2005 Nov 25;280(47):39002-15. doi: 10.1074/jbc.M508652200. Epub 2005 Sep 23. — View Citation
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Kaufman RJ. Orchestrating the unfolded protein response in health and disease. J Clin Invest. 2002 Nov;110(10):1389-98. doi: 10.1172/JCI16886. No abstract available. — View Citation
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Perlmutter DH. Liver injury in alpha1-antitrypsin deficiency: an aggregated protein induces mitochondrial injury. J Clin Invest. 2002 Dec;110(11):1579-83. doi: 10.1172/JCI16787. No abstract available. — View Citation
Seager Danciger J, Lutz M, Hama S, Cruz D, Castrillo A, Lazaro J, Phillips R, Premack B, Berliner J. Method for large scale isolation, culture and cryopreservation of human monocytes suitable for chemotaxis, cellular adhesion assays, macrophage and dendritic cell differentiation. J Immunol Methods. 2004 May;288(1-2):123-34. doi: 10.1016/j.jim.2004.03.003. — View Citation
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Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Evaluation of macrophage function. | From every study participant, we will collect blood from a vein through the placement of an intravenous catheter (IV). We will complete various experiments that will allow us to see how well each participant's macrophage cells are working. | On average, within 30 days from the time the blood is collected. | |
Secondary | Comparison of the amount of alpha-1 antitrypsin in the blood. | From every study participant, we will collect blood from a vein through an IV. | On average, within 30 days from the time the blood was collected. | |
Secondary | Comparison of the amount of an inflammatory marker in the blood, called C-reactive protein. | From every study participant, we will collect blood from a vein through and IV. | On average, within 30 days from the time the blood is collected. | |
Secondary | Evaluation of lung function. | Lung function testing will be done on every study participant. This is is done by forcefully blowing into a tube on at least 3 separate occasions. Albuterol, an inhaled medication used to expand lung airways, will be given after lung function testing and then the testing will be repeated after 30 minutes. | On average, within 30 days from the time the testing is completed. |
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