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ClinicalTrials.gov public records Last synced Sep 12, 2026, 8:27 AM EDT

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Showing 1–24 of 83 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha 1-Antitrypsin Deficiency Clear all
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 Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM EDT
Completed Phase 3 Interventional Results available

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

NCT00301366
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
38 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 Aug 18, 2014 · Synced Sep 12, 2026, 8:27 AM EDT
Enrolling by invitation Phase 3 Interventional

Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency

NCT02796937
View directory record Official record
Conditions
Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
Interventions
Alpha-1 MP
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
20 Years to 72 Years
Enrollment
290 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Wilmington, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Sep 12, 2026, 8:27 AM EDT
Recruiting No phase listed Observational

PiMZ Longitudinal Cohort (PiMZ Logic)

NCT06505603
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema or COPD
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 21, 2019 · Synced Sep 12, 2026, 8:27 AM EDT
Completed Phase 1 Interventional

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

NCT03815396
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, AATD
Interventions
INBRX-101/rhAAT-Fc
Drug
Lead sponsor
Inhibrx Biosciences, Inc
Industry
Eligibility
18 Years to 80 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
5
States / cities
Sacramento, California • Gainesville, Florida • Miami, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2022 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 28, 2023 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM EDT
Terminated No phase listed Observational

COPD Exacerbation Blood and Urine Biomarkers Study

NCT03823443
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease, Acute Exacerbation Copd, Alpha-1 Anti-trypsin Deficiency
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
35 Years to 80 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 3, 2024 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 Aug 10, 2026 · Synced Sep 12, 2026, 8:27 AM EDT
Terminated Phase 2 Interventional Results available

Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

NCT01379469
View directory record Official record
Conditions
Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
Interventions
Drug-Carbamazepine (Tegretol XR), Carbamazepine (Tegretol XR) Placebo
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
14 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced Sep 12, 2026, 8:27 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1, First-in-human Study of VX-668

NCT05727800
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
VX-668, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
114 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
5
States / cities
Tempe, Arizona • Gainesville, Florida • Orlando, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 14, 2024 · Synced Sep 12, 2026, 8:27 AM 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
4
States / cities
Boston, Massachusetts • Rochester, Minnesota • Charleston, South Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 12, 2026, 8:27 AM EDT
Recruiting No phase listed Observational

Alpha-1 Research Registry

NCT04157049
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Alpha-1 Foundation
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Coral Gables, Florida
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM 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 14, 2016 · Synced Sep 12, 2026, 8:27 AM 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 Sep 12, 2026, 8:27 AM EDT