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Showing 1–24 of 83 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha1-Antitrypsin Deficiency Clear all
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 4, 2026, 1:57 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 4, 2026, 1:57 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 4, 2026, 1:57 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 4, 2026, 1:57 PM EDT
Completed Phase 3 Interventional Results available

GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study

NCT02525861
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
GLASSIA
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
17
States / cities
Tucson, Arizona • Los Angeles, California • Sacramento, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2021 · Synced Sep 4, 2026, 1:57 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 12, 2018 · Synced Sep 4, 2026, 1:57 PM EDT
Completed Phase 2 Interventional Results available

Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency

NCT01213043
View directory record Official record
Conditions
Emphysema, Alpha 1-antitrypsin Deficiency (AATD)
Interventions
Prolastin-C, 60 mg/kg, Prolastin-C, 120 mg/kg
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 70 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
5
States / cities
Gainesville, Florida • Miami, Florida • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated May 19, 2013 · Synced Sep 4, 2026, 1:57 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 4, 2026, 1:57 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 28, 2023 · Synced Sep 4, 2026, 1:57 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 Sep 4, 2026, 1:57 PM 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 4, 2026, 1:57 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 4, 2026, 1:57 PM EDT
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 4, 2026, 1:57 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 14, 2016 · Synced Sep 4, 2026, 1:57 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 4, 2026, 1:57 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 Sep 4, 2026, 1:57 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 4, 2026, 1:57 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 4, 2026, 1:57 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 Sep 4, 2026, 1:57 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 Sep 4, 2026, 1:57 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 4, 2026, 1:57 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 4, 2026, 1:57 PM EDT
Active, not recruiting Phase 3 Interventional

Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)

NCT01983241
View directory record Official record
Conditions
Pulmonary Emphysema in Alpha-1 PI Deficiency
Interventions
Alpha-1 MP, 0.9% Sodium Chloride for Injection, USP
Biological · Other
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 70 Years
Enrollment
345 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
10
States / cities
Phoenix, Arizona • Gainesville, Florida • Leesburg, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Sep 4, 2026, 1:57 PM EDT
Active, not recruiting Phase 3 Interventional

An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease

NCT05899673
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Fazirsiran Injection
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2033
U.S. locations
5
States / cities
La Jolla, California • Redwood City, California • Gainesville, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Sep 4, 2026, 1:57 PM EDT