Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Vertex Pharmaceuticals Incorporated
VX-864 & Placebo
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18-35,35-65,65+
Liver Disease in Patients with Alpha-1 Antitrypsin Deficiency
RWTH Aachen University
Observational
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M,F
0-18,18-35,35-65,65+
Liver
MS
Phase 1 study to assess the safety, PK and PD of INBRX-101 in adults with Alpha-1 (rhAAT-Fc)
Inhibrx, Inc.
INBRX-101/rhAAT-Fc
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M,F
18-35,35-65,65+
MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Effects of different exercise training modalities in Alpha-1 Antitrypsin Deficiency
Schön Klinik Berchtesgadener Land
Observational
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M,F
35-65,65+
ZZ
A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease
Dicerna Pharmaceuticals, Inc.
DCR-A1AT & placebo
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,M,F
18-35,35-65,65+
Liver
ZZ
Alvelestat (MPH996) for the Treatment of ALpha-1 ANTitrypsin Deficiency
University of Alabama at Birmingham
Alvelestat & Placebo
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M,F
18-35,35-65,65+
SZ,ZZ
Alpha-1 Research Registry
Alpha-1 Foundation
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M,F
0-18,18-35,35-65,65+
Liver,Lung
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis
Medical University of South Carolina
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,M,F
18-35,35-65,65+
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
COPD Exacerbation Blood and Urine Biomarkers Study
Columbia University Medical Center
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M,F
35-65,65+
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
University of Florida
Albuterol inhaler
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M,F
18-35,35-65,65+
Lung
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease
McMaster University
Hyperpolarized 129Xe & MRI
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M,F
18-35,35-65,65+
Lung
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AAT
Arrowhead Pharmaceuticals
ARO-AAT Injection
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M,F
18-35,35-65,65+
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT
Arrowhead Pharmaceuticals
ARO-AAT Injection & Placebo
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M,F
18-35,35-65,65+
Liver
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.
Mereo BioPharma
Alvelestat oral tablet & Placebo
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M,F
18-35,35-65,65+
Z,M,S,ZZ
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Vertex Pharmaceuticals Incorporated
Vx-814 & Placebo
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M,F
18-35,35-65,65+
ZZ
The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft‑Versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant
CSL Behring
Alpha-1 Proteinase inhibitor & Placebo
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M,F
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Evaluating the Genetic Causes and Progression of Cholestatic Liver Diseases (LOGIC)
Arbor Research Collaborative for Health
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M,F
0-18,18-35
Liver
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
Grifols Therapeutics LLC
Prolastin-C & Placebo
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M,F
18-35,35-65,65+
Z,S,SZ,ZZ
Alpha-1 Antitrypsin Deficiency Adult liver study
St. Louis University
Liver Biopsy
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M,F
0-18,18-35,35-65,65+
Liver
ZZ
Treatment of GVHD in Hematopoietic Stem Cell Transplant (HSCT) Recipients Using AAT Plus Corticosteroids (CS) Compared With Corticosteroids Alone
CSL Behring
Alpha-1 proteinase inhibitor & Placebo
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M,F
18-35,35-65,65+
Z,M,S,MS,MZ,MF,SS,SZ,ZZ,FZ,FF
Earco Registry. History of Patients with Alpha-1 Antitrypsin
Hospital Universitari Vall d'Hebron Research Institute
None
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M,F
0-18,18-35,35-65,65+
SZ,ZZ
Evaluate Efficacy and Safety of "Kamada-AAT for inhalation" in Patients with AATD
Kamada, Ltd.
Placebos
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M,F
18-35,35-65
ZZ