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AATD clinical trials

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NCT ID: NCT03815396 Recruiting - Clinical trials for Alpha-1 Antitrypsin Deficiency

Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency

Start date: July 19, 2019
Phase: Phase 1
Study type: Interventional

This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD) administrations of INBRX-101. The planned dosing schedule is IV every 3 to 4 weeks.

NCT ID: NCT01851642 Recruiting - Clinical trials for Alpha-1 Antitrypsin Deficiency

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

Start date: September 2007
Study type: Observational

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.

NCT ID: NCT01832220 Completed - AATD Clinical Trials

Genomic Research in Alpha-1 Antitrypsin Deficiency

GRADS Alpha-1
Start date: May 2013
Phase: N/A
Study type: Observational

This project is designed to examine the interaction between the microflora in the lower airway and the concentration of a serum protein called alpha-1 antitrypsin. The hypothesis is that alpha-1 antitrypsin impacts the diversity and content of the lower airway microflora, resulting in a less inflammatory airway. The Specific Aims are: 1. To compare the lower respiratory tract microbiome and virome population diversity and content in age and GOLD stage matched PiZZ individuals not receiving augmentation therapy, PiZZ individuals on augmentation therapy, PiMZ individuals not receiving augmentation therapy, and PiMM individuals with chronic obstructive pulmonary disease (COPD). 2. Determine correlations between bronchoalveolar lavage (BAL) and peripheral blood gene expression patterns and patterns in lung microbial and viral populations across all cohorts. 3. Correlate the presence or absence of computed tomography (CT) bronchiectasis and bronchiolectasis with patterns in the microbiome population diversity and content. 4. To identify and define novel molecular phenotypes of Alpha-1 Antitrypsin Deficiency (AATD) based on computational integration of clinical, transcriptomic, and microbiome data.

NCT ID: NCT01810458 Completed - Liver Fibrosis Clinical Trials

Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

Start date: October 2013
Study type: Observational

We hypothesize that individuals with Alpha-1 Antitrypsin (AAT) deficiency have ongoing liver injury which is not detected by the usual blood tests used to look at liver function. This ongoing liver injury leads to cirrhosis in a significant number of adults with AAT deficiency.