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Showing 1–24 of 85 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha1 Anti-Trypsin Deficiency Clear all
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 10, 2026, 9:30 AM 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 10, 2026, 9:30 AM 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 4, 2026 · Synced Sep 10, 2026, 9:30 AM EDT
Completed Phase 4 Interventional Results available

Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency

NCT00261833
View directory record Official record
Conditions
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Interventions
Alpha1-proteinase inhibitor, Placebo
Biological · Other
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 65 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
4
States / cities
Denver, Colorado • Miami, Florida • Hershey, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2015 · Synced Sep 10, 2026, 9:30 AM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Study of OsrhAAT or Placebo in Healthy Volunteers

NCT05315921
View directory record Official record
Conditions
Emphysema Secondary to Congenital AATD
Interventions
OsrhAAT 1 mg/kg IV, OsrhAAT 3 mg/kg IV, OsrhAAT 10 mg/kg IV, OsrhAAT 20 mg/kg IV, OsrhAAT 40 mg/kg IV, OsrhAAT 60 mg/kg IV
Drug
Lead sponsor
Healthgen Biotechnology Corp.
Industry
Eligibility
18 Years to 55 Years
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
1
States / cities
Cypress, California
Source: ClinicalTrials.gov public record
Updated Dec 11, 2022 · Synced Sep 10, 2026, 9:30 AM EDT
Completed No phase listed Observational

Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)

NCT01810458
View directory record Official record
Conditions
Liver Fibrosis, Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD
Interventions
Abdominal ultrasound, History and physical, Intravenous catheter, Blood draw, Liver questionnaire, Liver Biopsy, Midazolam, Fentanyl, Lidocaine, Acetaminophen, Lorazepam, Oxycodone/Acetaminophen, Ondansetron
Device · Procedure · Other + 1 more
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 70 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 20, 2023 · Synced Sep 10, 2026, 9:30 AM EDT
Completed Early Phase 1 Interventional

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

NCT00377416
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV2-CB-hAAT Gene Vector
Genetic
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2020
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 9, 2020 · Synced Sep 10, 2026, 9:30 AM 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 10, 2026, 9:30 AM 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 10, 2026, 9:30 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 10, 2026, 9:30 AM 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 10, 2026, 9:30 AM 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 Sep 10, 2026, 9:30 AM EDT
Withdrawn Phase 2 Interventional

A Research Trial of Aralast NP in New Onset Diabetes (RETAIN) - Part II

NCT01183455
View directory record Official record
Conditions
Diabetes Mellitus, Type 1
Interventions
Aralast NP, Placebo
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
15
States / cities
La Jolla, California • Aurora, Colorado • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2014 · Synced Sep 10, 2026, 9:30 AM 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 7, 2025 · Synced Sep 10, 2026, 9:30 AM 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 10, 2026, 9:30 AM 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 16, 2007 · Synced Sep 10, 2026, 9:30 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 10, 2026, 9:30 AM EDT
Completed Phase 2 Interventional Results available

Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis

NCT01684410
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Alpha-1 HC 100 mg, Placebo, Alpha-1 HC 200 mg
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Birmingham, Alabama • Denver, Colorado • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2016 · Synced Sep 10, 2026, 9:30 AM 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 22, 2018 · Synced Sep 10, 2026, 9:30 AM 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 Sep 10, 2026, 9:30 AM EDT
Completed No phase listed Observational

AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry

NCT01974830
View directory record Official record
Conditions
Alpha-1 Anti-trypsin Deficiency
Interventions
Not listed
Lead sponsor
Coram Clinical Trials
Other
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Centennial, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 22, 2019 · Synced Sep 10, 2026, 9:30 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 10, 2026, 9:30 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 10, 2026, 9:30 AM 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 10, 2026, 9:30 AM EDT