Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

Related Clinical Trial
A Phase 1, First-in-human Study of VX-668 Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype A Phase 1, First-in-human Study of VX-634 A Phase I of OsrhAAT in Healthy Volunteers Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease A Study of Belcesiran in Patients With A1ATD-Associated Liver Disease A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiMZ Subjects ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals COPD Exacerbation Blood and Urine Biomarkers Study Alpha-1 Carrier Genomics Study Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype Study of ARO-AAT in Normal Adult Volunteers Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry Respreeza® Self-administration and Learning Program (AmAREtTI Study) Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Environment Effect on Six-Minute Walk Test Performance Alpha-1 Foundation DNA and Tissue Bank Alpha1-antitrypsin Deficiency Registry Alpha-1 Research Registry Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency Alpha-1 Coded Testing(ACT) Study Long-Term Follow-up Study of ADVM-043 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation” EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency Alpha-1 Foundation Research Registry Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients Evaluate Efficacy and Safety of “Kamada-AAT for Inhalation” in Patients With AATD A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 – Proteinase Inhibitor) Aralast alpha1-proteinase Inhibitor Surveillance Study Pharmacokinetic Study of ARALAST (Human Alpha1- PI) The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

Brief Title

Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

Official Title

The Role of Conformational Diseases on Macrophage Function

Brief Summary

      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.
    

Detailed Description

      AAT deficiency is a genetic disorder that affects around 100,000 people in the USA, including
      1-3% of all people diagnosed with chronic obstructive pulmonary disease (COPD). In AAT
      deficient people diagnosed with COPD, it was originally believed the cause of the disease was
      due to a lack of supply of alpha-1 antitrypsin. However, early information gathered in our
      laboratory suggests another cause of the development of COPD and the progressing of the
      disease may be due to a malfunction in macrophages.

      CF is also a genetic disorder which affects 1/300 births among the Caucasian population. One
      of the main symptoms of CF is inflammation of the lung tissue. Lung macrophages play a major
      role in lung inflammation as well as in helping to resolve the inflammation.

      Inflammation is an important defense of the body. It is the body's response to infection
      causing germs and things that may cause irritation, as well as, a way for the body to repair
      damaged tissue.

      We suggest that the effects of AAT deficiency and CF decreases the inflammation response in
      the lungs and also restricts the ability of macrophages to correct that inflammation once it
      occurs.
    


Study Type

Observational


Primary Outcome

Evaluation of macrophage function.

Secondary Outcome

 Comparison of the amount of alpha-1 antitrypsin in the blood.

Condition

Alpha-1 Antitrypsin Deficiency

Intervention

History and physical exam.

Study Arms / Comparison Groups

 AAT Deficiency
Description:  Those diagnosed with Alpha-1 Antitrypsin (AAT) Deficiency. At every study visit, a history and physical exam (H&P), blood draw, and pulmonary function testing (PFTs) with the use of an albuterol inhaler will be done.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Procedure

Estimated Enrollment

220

Start Date

August 9, 2007

Completion Date

July 20, 2023

Primary Completion Date

July 20, 2023

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Mark Brantly, MD, 352-273-8666, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01851642

Organization ID

IRB201501051

Secondary IDs

699

Responsible Party

Sponsor

Study Sponsor

University of Florida


Study Sponsor

Mark Brantly, MD, Principal Investigator, University of Florida, College of Medicine, Division of Pulmonary, Critical Care, and Sleep Medicine


Verification Date

September 2022