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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
Completed No phase listed Observational

Genomic Research in Alpha-1 Antitrypsin Deficiency

NCT01832220
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency, AATD
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
40 Years to 80 Years
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
9
States / cities
Tucson, Arizona • San Francisco, California • Denver, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2016 · Synced Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 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 21, 2019 · Synced Sep 27, 2026, 2:14 PM 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 27, 2026, 2:14 PM EDT
Completed Phase 1 Interventional

The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes

NCT01319331
View directory record Official record
Conditions
Diabetes, Type 1 Diabetes
Interventions
Alpha 1-Antitrypsin (AAT, Aralast NP)
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
6 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 23, 2017 · Synced Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 PM EDT
Terminated Phase 1 Interventional Results available

A Research Trial of Aralast in New Onset Diabetes (RETAIN)

NCT01183468
View directory record Official record
Conditions
Diabetes Mellitus, Type 1
Interventions
Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 35 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
15
States / cities
San Diego, California • Aurora, Colorado • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2018 · Synced Sep 27, 2026, 2:14 PM EDT
Active, not recruiting Phase 1 Interventional

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

NCT06738017
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
BMN 349, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 75 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
4
States / cities
San Diego, California • St Louis, Missouri • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 PM 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 27, 2026, 2:14 PM EDT
Completed No phase listed Observational

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

NCT02922751
View directory record Official record
Conditions
Biliary Atresia, Alagille Syndrome, Alpha1 Anti-Trypsin Deficiency, Portal Hypertension, Liver Fibrosis, Cholestasis
Interventions
Liver Stiffness Measurement (LSM)
Other
Lead sponsor
Arbor Research Collaborative for Health
Other
Eligibility
Up to 21 Years
Enrollment
552 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2024 · Synced Sep 27, 2026, 2:14 PM EDT
Completed Phase 2 Interventional Results available

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

NCT01054339
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
rAAV1-CB-hAAT
Drug
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
3
States / cities
Denver, Colorado • Worcester, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 27, 2019 · Synced Sep 27, 2026, 2:14 PM 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 27, 2026, 2:14 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 Sep 27, 2026, 2:14 PM EDT
Completed Phase 2 Interventional

4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?

NCT00067756
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
4-PBA
Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 2, 2014 · Synced Sep 27, 2026, 2:14 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 Aug 10, 2026 · Synced Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 PM EDT
Terminated Phase 2 Interventional Results available

A Study of Belcesiran in Patients With AATLD

NCT04764448
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Belcesiran, Placebo
Drug · Other
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
18 Years to 75 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
3
States / cities
La Jolla, California • Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Sep 27, 2026, 2:14 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 Sep 27, 2026, 2:14 PM EDT
Completed Phase 2 Interventional

Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency

NCT03114020
View directory record Official record
Conditions
Emphysema, Alpha 1-Antitrypsin Deficiency
Interventions
Hyaluronic Acid Inhalation Solution, Placebo Inhalation Solution
Drug
Lead sponsor
Gerard Turino
Individual
Eligibility
18 Years to 80 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
5
States / cities
Miami, Florida • Fayetteville, New York • Charleston, South Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2020 · Synced Sep 27, 2026, 2:14 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 Sep 9, 2026 · Synced Sep 27, 2026, 2:14 PM EDT