Clinical trials: Alpha-1-antitrypsin Deficiency
Emphysema, Lung Diseases, Obstructive
Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals
Alpha 1-antitrypsin-deficient individuals develop severe destructive lung disease much earlier and their lung function declines faster than the general population of individuals with chronic obstructive lung disease.
- Conditions
- Emphysema, Lung Diseases, Obstructive, Alpha 1-Antitrypsin Deficiency
- Trial name
- NCT00001462
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Alpha-1 Antitrypsin Deficiency, AATD
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
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)…
- Trial name
- rhAAT-Fc (NCT03815396)
- Testing
- INBRX-101/rhAAT-Fc
- Where
- 10 sites in United States, New Zealand and 1 more
- Volunteers
- 31 people
- Sponsor
- Inhibrx Biosciences, Inc
Alpha 1-Antitrypsin Deficiency
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1…
- Trial name
- ChAMP (NCT00295061)
- Testing
- Alpha-1 MP, alpha-1 proteinase inhibitor (human)
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Emphysema or COPD, Aneurysm
The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin Degradation
Background: Elastin is a unique protein providing elasticity, resilience and deformability to dynamic tissues, such as lungs and vasculature. Elastin fibers are characterized by their high affinity for calcium.
- Conditions
- Emphysema or COPD, Aneurysm, Cystic Fibrosis, Alpha 1-Antitrypsin Deficiency
- Trial name
- NCT03285100
- Testing
- Venipuncture
- Where
- Netherlands
- Volunteers
- 30 people
- Sponsor
- Canisius-Wilhelmina Hospital
Alpha-1 Antitrypsin Deficiency, Emphysema
Alpha-1 Carrier Genomics Study
The goal of this study is to better understand why some Alpha-1 genotype MZ (PiMZ) individuals develop chronic obstructive pulmonary disease (COPD) while others do not.
- Conditions
- Alpha-1 Antitrypsin Deficiency, Emphysema, COPD, Smoking
- Trial name
- NCT02810327
- Testing
- Genetic Sequencing
- Where
- United States
- Volunteers
- 117 people
- Sponsor
- Medical University of South Carolina
Alpha 1-Antitrypsin Deficiency
Alpha-1 Foundation Research Registry
The Alpha-1 Foundation Research Registry is a confidential database made up of individuals diagnosed with severe alpha-1 antitrypsin deficiency (Alpha-1) or the carrier state.
- Trial name
- NCT00499941
- Where
- United States
- Volunteers
- 6,655 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Alpha 1-Antitrypsin Deficiency
4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.
- Trial name
- NCT00067756
- Testing
- 4-PBA
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
Alpha 1 Antitrypsin Deficiency
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.
- Trial name
- RepAIR1 (NCT07431112)
- Testing
- AIR-001 SAD dose level 1, AIR-001 SAD dose level 2, AIR-001 SAD dose level 3
- Where
- 13 sites in Australia, Georgia and 7 more
- Volunteers
- 54 people
- Sponsor
- AIRNA Corporation
Type 1 Diabetes Mellitus
Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus
This is a multicenter, randomized, partial-blinded, five-arm, placebo-controlled study of human plasma-derived alpha1-proteinase inhibitor (alpha1-PI) in children (ages 6-11 years old) and teens/adults (ages 12-35 years old) with…
- Trial name
- NCT02093221
- Testing
- 180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI
- Where
- 36 sites in United States
- Volunteers
- 76 people
- Sponsor
- Grifols Therapeutics LLC
Alpha 1-Antitrypsin, COPD
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function…
- Conditions
- Alpha 1-Antitrypsin, COPD, Antibody Deficiency
- Trial name
- IgG in AATD (NCT07135427)
- Testing
- 20-valent pneumococcal conjugate vaccine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Alabama at Birmingham
Alpha1-Antitrypsin Deficiency
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of AAT, called Z-AAT.
- Trial name
- ALPHATUDE (NCT06512454)
- Testing
- No Intervention
- Where
- 8 sites in United States, Austria and 4 more
- Volunteers
- 500 people
- Sponsor
- Takeda
Covid19
Alpha-1-Antitrypsin-Deficiency in COVID-19
The blood concentrations of alpha-1-antitrypsin are monitored during COVID-19. The clinical course of patients that received AAT infusion for clinical indication is monitored.
- Trial name
- CORSAAR-AAT (NCT04799873)
- Testing
- AAT( Alpha 1 Antitrypsin)
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Universität des Saarlandes
Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha 1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to assess the safety of the treatment in subjects with AAT…
- Trial name
- NCT02001688
- Testing
- Kamada-AAT for Inhalation, 80mg, Kamada-AAT for Inhalation, 160mg
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Kamada, Ltd.
Alpha1 Anti-Trypsin Deficiency
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiXZ Subjects
This study is composed of two parts. Part A: will test single doses of ZF874 in a double-blind, randomised, placebo-controlled and dose-escalating design (except Group 7, which will be open-label and without placebo).
- Trial name
- NCT04443192
- Testing
- ZF874
- Where
- 5 sites in United Kingdom
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Z Factor Limited
Alpha-1 Antitrypsin Deficiency (AATD)
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults…
- Trial name
- NCT07227207
- Testing
- TSRA-196
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 72 people
- Sponsor
- Tessera Therapeutics, Inc.
Alpha-1 Antitrypsin Deficiency, Emphysema
Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency
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…
- Conditions
- Alpha-1 Antitrypsin Deficiency, Emphysema, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT03008915
- Testing
- Aspirin, Withdrawal from alpha1 antitrypsin replacement therapy
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Columbia University
alpha1-antitrypsin Deficiency
Liver Disease in Patients With alpha1-antitrypsin Deficiency
Alpha1-antitrypsin deficiency (AATD) is the third most common genetic disorder leading to death worldwide. Apart from lung disease, AATD also leads to liver involvement in up to 50% of patients.
- Trial name
- NCT02929940
- Testing
- No intervention
- Where
- Germany
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- RWTH Aachen University
Alpha-1-Antitrypsin Deficiency, Chronic Obstructive Lung Disease
Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency
The objective of this study is to evaluate the safety and effectiveness of the RePneu Lung Volume Reduction Coil (LVRC) in patients with Alpha-1-Antitrypsin deficiency (AATD) caused emphysema.
- Conditions
- Alpha-1-Antitrypsin Deficiency, Chronic Obstructive Lung Disease, Emphysema
- Trial name
- LuReCAA (NCT02273349)
- Testing
- Lung Volume Reduction Coil (PneumrX Inc.)
- Where
- 2 sites in Denmark, Germany
- Sponsor
- University Hospital, Saarland
Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency
Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)
This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density…
- Conditions
- Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema
- Trial name
- NCT07326592
- Testing
- CE1226
- Where
- 2 sites in Denmark
- Volunteers
- 270 people
- Sponsor
- CSL Behring
Retrospective Cohort of Patients Diagnosed With HCC on Non-Cirrhotic Liver
Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer
Patients with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease, have a 20- to 50-fold higher risk of developing primary liver cancer (PLC), such as hepatocellular carcinoma (HCC), in both cirrhotic and non-cirrhotic…
- Trial name
- CHAMAD (NCT07145385)
- Testing
- multiplex digital PCR
- Where
- France
- Volunteers
- 71 people
- Sponsor
- University Hospital, Bordeaux