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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha 1-Antitrypsin Deficiency Clear all
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 3, 2026 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 1Phase 2 Interventional

Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

NCT00161707
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
Aerosolized, Recombinant Alpha 1-Antitrypsin
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
4
States / cities
Denver, Colorado • Gainesville, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 4, 2021 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, First-in-human Study of VX-634

NCT05579431
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
VX-634, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
127 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
5
States / cities
Tempe, Arizona • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2024 · Synced Jul 21, 2026, 9:24 PM EDT
Terminated Not applicable Interventional

Environment Effect on Six-Minute Walk Test Performance

NCT02502201
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease, Alpha-1 Antitrypsin Deficiency
Interventions
six-minute walk study indoors, six-minute walk study outdoors
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 21, 2019 · Synced Jul 21, 2026, 9:24 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 15, 2026 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 2 Interventional Results available

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

NCT04474197
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
VX-864, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
22
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 19 more
Source: ClinicalTrials.gov public record
Updated May 25, 2022 · Synced Jul 21, 2026, 9:24 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 18, 2012 · Synced Jul 21, 2026, 9:24 PM EDT
Completed No phase listed Observational

The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency

NCT03039335
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Biocerna LLC
Industry
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Fulton, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 15, 2018 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 3 Interventional Results available

Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults

NCT00295061
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Alpha-1 MP, alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
8
States / cities
Denver, Colorado • Gainesville, Florida • Miami, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2014 · Synced Jul 21, 2026, 9:24 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

NCT07227207
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency (AATD)
Interventions
TSRA-196
Drug
Lead sponsor
Tessera Therapeutics, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
Boston, Massachusetts • Charleston, South Carolina • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Jul 21, 2026, 9:24 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 10, 2025 · Synced Jul 21, 2026, 9:24 PM EDT
Terminated Phase 2 Interventional Results available

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

NCT04167345
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
VX-814, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
22
States / cities
Birmingham, Alabama • Sacramento, California • Denver, Colorado + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2022 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 2 Interventional Results available

Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"

NCT02001688
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Kamada-AAT for Inhalation, 80mg, Placebo, Kamada-AAT for Inhalation, 160mg
Drug
Lead sponsor
Kamada, Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
2
States / cities
Gainesville, Florida • Tyler, Texas
Source: ClinicalTrials.gov public record
Updated Jan 17, 2020 · Synced Jul 21, 2026, 9:24 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study

NCT00500123
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Alpha-1 Antitrypsin Genotype
Procedure
Lead sponsor
University of Florida
Other
Eligibility
Not listed
Enrollment
50,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2050
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 23, 2025 · Synced Jul 21, 2026, 9:24 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Alpha-1 Foundation Research Registry

NCT00499941
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Medical University of South Carolina
Other
Eligibility
Not listed
Enrollment
6,655 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2021
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Mar 2, 2021 · Synced Jul 21, 2026, 9:24 PM EDT
Completed No phase listed Observational Results available

Long-Term Follow-up Study of ADVM-043

NCT03804021
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
No Intervention
Genetic
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
2
States / cities
Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 2 Interventional Results available

Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.

NCT01669421
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Alpha-1 Antitrypsin (human)
Drug
Lead sponsor
Michael Campos, MD
Other
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated May 7, 2019 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 1Phase 2 Interventional

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

NCT04722887
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 80 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Miami, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 11, 2025 · Synced Jul 21, 2026, 9:24 PM EDT
Recruiting Phase 3 Interventional

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

NCT05677971
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Fazirsiran Injection, Placebo
Drug · Other
Lead sponsor
Takeda
Industry
Eligibility
18 Years to 75 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
37
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 28 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 21, 2026, 9:24 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT06389877
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
BEAM-302
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Birmingham, Alabama • Boston, Massachusetts • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Jul 21, 2026, 9:24 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 3, 2008 · Synced Jul 21, 2026, 9:24 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 Nov 9, 2025 · Synced Jul 21, 2026, 9:24 PM EDT
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 21, 2025 · Synced Jul 21, 2026, 9:24 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

NCT03945292
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Fazisiran Injection, Placebo
Drug · Other
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
16
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Jul 21, 2026, 9:24 PM EDT