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ClinicalTrials.gov public records Last synced Sep 30, 2026, 7:09 AM EDT

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Showing 1–24 of 82 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Alpha-1 Antitrypsin Deficiency (AATD) Clear all
Recruiting No phase listed Observational

A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems

NCT06512454
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
No Intervention
Other
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
3
States / cities
Gainesville, Florida • Charleston, South Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Sep 30, 2026, 7:09 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 11, 2026 · Synced Sep 30, 2026, 7:09 AM 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 6, 2026 · Synced Sep 30, 2026, 7:09 AM 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 15, 2026 · Synced Sep 30, 2026, 7:09 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
5
States / cities
Jacksonville, Florida • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Sep 30, 2026, 7:09 AM EDT
Terminated Phase 3 Interventional Results available

Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency

NCT02722304
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Interventions
ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg, GLASSIA 120 mg/kg, Human Albumin 2%
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
14
States / cities
Peoria, Arizona • Newport Beach, California • Kissimmee, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated May 13, 2021 · Synced Sep 30, 2026, 7:09 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 15, 2016 · Synced Sep 30, 2026, 7:09 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 4, 2024 · Synced Sep 30, 2026, 7:09 AM 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 16, 2026 · Synced Sep 30, 2026, 7:09 AM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Therapy to Elevate CD4 Counts in HIV-1 Disease

NCT01370018
View directory record Official record
Conditions
HIV Disease
Interventions
alpha-1-Proteinase Inhibitor
Biological
Lead sponsor
Institute for Human Genetics and Biochemistry
Other
Eligibility
30 Years to 70 Years · Male only
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2007
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2021 · Synced Sep 30, 2026, 7:09 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 8, 2019 · Synced Sep 30, 2026, 7:09 AM 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 27, 2026 · Synced Sep 30, 2026, 7:09 AM EDT
Completed Phase 2 Interventional Results available

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

NCT03679598
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
Interventions
Alvelestat (MPH966), Placebo
Drug · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 80 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Sep 30, 2026, 7:09 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 18, 2020 · Synced Sep 30, 2026, 7:09 AM 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 17, 2021 · Synced Sep 30, 2026, 7:09 AM 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 10, 2025 · Synced Sep 30, 2026, 7:09 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 19, 2014 · Synced Sep 30, 2026, 7:09 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 23, 2018 · Synced Sep 30, 2026, 7:09 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 3, 2021 · Synced Sep 30, 2026, 7:09 AM EDT
Terminated Phase 4 Interventional Results available

Aralast alpha1-proteinase Inhibitor Surveillance Study

NCT00313144
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
ARALAST Alpha1-Proteinase Inhibitor
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
San Marino, California
Source: ClinicalTrials.gov public record
Updated May 26, 2021 · Synced Sep 30, 2026, 7:09 AM 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 26, 2022 · Synced Sep 30, 2026, 7:09 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 29, 2023 · Synced Sep 30, 2026, 7:09 AM 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 22, 2019 · Synced Sep 30, 2026, 7:09 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 13, 2026 · Synced Sep 30, 2026, 7:09 AM EDT