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ClinicalTrials.gov public records Last synced Oct 30, 2026, 4:47 PM EDT

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Showing 49–72 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Alpha-1 Protease Inhibitor Deficiency Clear all
Recruiting Phase 1 Interventional

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

NCT06049082
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
KB408 (Nebulization)
Drug
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Gainesville, Florida • Charleston, South Carolina • The Woodlands, Texas
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Oct 30, 2026, 4:47 PM EDT
Completed Phase 2Phase 3 Interventional

Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency

NCT00460096
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Kamada-API
Drug
Lead sponsor
Kamada, Ltd.
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007
U.S. locations
3
States / cities
Denver, Colorado • Gainesville, Florida • Tyler, Texas
Source: ClinicalTrials.gov public record
Updated Oct 17, 2007 · Synced Oct 30, 2026, 4:47 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCT02168686
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
ADVM-043
Genetic
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 5, 2023 · Synced Oct 30, 2026, 4:47 PM EDT
Terminated Not applicable Interventional

Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant Suitability

NCT01241942
View directory record Official record
Conditions
Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis, Bronchiectasis, Sarcoidosis, Pulmonary Hypertension, Alpha-1 Antitrypsin Deficiency
Interventions
Steen Solution™, Conventional Lung Transplant
Device · Other
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2018 · Synced Oct 30, 2026, 4:47 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis

NCT02947087
View directory record Official record
Conditions
Chronic Pancreatitis
Interventions
Prolastin-C, Placebo
Drug
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Jun 29, 2023 · Synced Oct 30, 2026, 4:47 PM EDT
Terminated Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Safety and Efficacy of (α1Proteinase Inhibitor, α1PI) in HIV Disease

NCT01731691
View directory record Official record
Conditions
HIV Disease
Interventions
α1 Proteinase Inhibitor, Placebos
Biological · Drug
Lead sponsor
Institute for Human Genetics and Biochemistry
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 30, 2026, 4:47 PM EDT
Terminated Phase 4 Interventional Results available

Aralast alpha1-proteinase Inhibitor Surveillance Study

NCT00313144
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
ARALAST Alpha1-Proteinase Inhibitor
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
San Marino, California
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Oct 30, 2026, 4:47 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Alpha-1 Foundation DNA and Tissue Bank

NCT00884455
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
0 Years to 100 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2033
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed No phase listed Observational

Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD)

NCT01419158
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
Interventions
Not listed
Lead sponsor
Alpha-1 Foundation
Other
Eligibility
18 Years and older
Enrollment
3,457 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
18
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 19, 2012 · Synced Oct 30, 2026, 4:47 PM EDT
Suspended No phase listed Observational

Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)

NCT00571272
View directory record Official record
Conditions
Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Arbor Research Collaborative for Health
Other
Eligibility
Up to 25 Years
Enrollment
1,675 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2029
U.S. locations
16
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Recruiting Phase 4 Interventional

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

NCT07135427
View directory record Official record
Conditions
Alpha 1-Antitrypsin, COPD, Antibody Deficiency
Interventions
20-valent pneumococcal conjugate vaccine
Biological
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

NCT05643495
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
VX-864
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
10
States / cities
Gainesville, Florida • Orlando, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2025 · Synced Oct 30, 2026, 4:47 PM EDT
Active, not recruiting No phase listed Observational

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

NCT05297812
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Genetic Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
286 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed No phase listed Observational Results available

Alpha-1 Carrier Genomics Study

NCT02810327
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Emphysema, COPD, Smoking
Interventions
Genetic Sequencing
Genetic
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Jul 22, 2019 · Synced Oct 30, 2026, 4:47 PM EDT
Recruiting Phase 3 Interventional

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

NCT06165341
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Fazirsiran Injection, Placebo
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
20
States / cities
Phoenix, Arizona • Tucson, Arizona • La Jolla, California + 15 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed Phase 1 Interventional Results available

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency

NCT00430768
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV1-CB-hAAT
Biological
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 15, 2016 · Synced Oct 30, 2026, 4:47 PM EDT
Active, not recruiting No phase listed Observational

Alpha-1 Antitrypsin Deficiency Adult Liver Study

NCT02014415
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Liver Biopsy (Biopsy Group Only)
Procedure
Lead sponsor
St. Louis University
Other
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
3
States / cities
San Diego, California • Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Role of Genetic Factors in the Development of Lung Disease

NCT00001532
View directory record Official record
Conditions
Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma, Pulmonary Sarcoidosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 90 Years
Enrollment
3,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1996
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals

NCT00001462
View directory record Official record
Conditions
Emphysema, Lung Diseases, Obstructive, Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 4:47 PM EDT
Recruiting Phase 1 Interventional

Gene Therapy for Alpha 1- Antitrypsin Deficiency

NCT06996756
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
AAV8hAAT(AVL)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed Phase 2 Interventional

Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

NCT05856331
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema
Interventions
SAR447537, Zemaira
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Oct 30, 2026, 4:47 PM EDT
Recruiting Phase 3 Interventional

A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)

NCT07555483
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Alpha-1 15%, Liquid Alpha1-PI
Drug · Other
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
10
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 30, 2026, 4:47 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency

NCT02691611
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years to 85 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2020
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 6, 2025 · Synced Oct 30, 2026, 4:47 PM EDT
Completed No phase listed Observational

The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency

NCT00532805
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
7
States / cities
Denver, Colorado • Gainesville, Florida • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jan 23, 2018 · Synced Oct 30, 2026, 4:47 PM EDT