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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha1-proteinase Inhibitor Deficiency Clear all
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 Jul 23, 2026, 12:48 PM EDT
Active, not recruiting No phase listed Observational

Alpha-1 Antitrypsin Deficiency Adult Liver Study

NCT02014415
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency
Interventions
Liver Biopsy (Biopsy Group Only)
Procedure
Lead sponsor
St. Louis University
Other
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2027
U.S. locations
3
States / cities
San Diego, California • Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jul 23, 2026, 12:48 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 23, 2026, 12:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCT02168686
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
ADVM-043
Genetic
Lead sponsor
Adverum Biotechnologies, Inc.
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Gainesville, Florida • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Jul 23, 2026, 12:48 PM EDT
Completed Phase 2 Interventional Results available

Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency

NCT03008915
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Emphysema, Chronic Obstructive Pulmonary Disease
Interventions
Aspirin, Placebo, Withdrawal from alpha1 antitrypsin replacement therapy
Drug · Other
Lead sponsor
Columbia University
Other
Eligibility
40 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jul 23, 2026, 12:48 PM EDT
Recruiting Phase 1 Interventional

Gene Therapy for Alpha 1- Antitrypsin Deficiency

NCT06996756
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
AAV8hAAT(AVL)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Jul 23, 2026, 12:48 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 23, 2026, 12:48 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 Jul 23, 2026, 12:48 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 23, 2026, 12:48 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 23, 2026, 12:48 PM EDT
Completed No phase listed Observational

Genomic Research in Sarcoidosis

NCT01831739
View directory record Official record
Conditions
Sarcoidosis
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 85 Years
Enrollment
368 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 Jul 23, 2026, 12:48 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 23, 2026, 12:48 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 23, 2026, 12:48 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 Jul 23, 2026, 12:48 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 23, 2026, 12:48 PM 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 Jul 23, 2026, 12:48 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 Jul 23, 2026, 12:48 PM EDT
Terminated Phase 2 Interventional Results available

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

NCT02093221
View directory record Official record
Conditions
Type 1 Diabetes Mellitus
Interventions
180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI, Placebo
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
6 Years to 35 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
36
States / cities
Tucson, Arizona • Los Angeles, California • Orange, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2018 · Synced Jul 23, 2026, 12:48 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Alpha-1 Foundation DNA and Tissue Bank

NCT00884455
View directory record Official record
Conditions
Alpha 1 Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
0 Years to 100 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2033
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Jul 23, 2026, 12:48 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 Jul 12, 2026 · Synced Jul 23, 2026, 12:48 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 23, 2026, 12:48 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 Jul 23, 2026, 12:48 PM 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 Jul 23, 2026, 12:48 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 23, 2026, 12:48 PM EDT