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ClinicalTrials.gov public records Last synced Sep 8, 2026, 4:35 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: AATD Clear all
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 Sep 8, 2026, 4:35 PM EDT
Active, not recruiting Phase 2 Interventional

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

NCT05897424
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema
Interventions
SAR447537
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 80 Years
Enrollment
185 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
18
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Sep 8, 2026, 4:35 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 8, 2026, 4:35 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 8, 2026, 4:35 PM 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 8, 2026, 4:35 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 8, 2026, 4:35 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 8, 2026, 4:35 PM EDT
Completed Phase 1Phase 2 Interventional

Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency

NCT00161707
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
Aerosolized, Recombinant Alpha 1-Antitrypsin
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003
U.S. locations
4
States / cities
Denver, Colorado • Gainesville, Florida • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 4, 2021 · Synced Sep 8, 2026, 4:35 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 8, 2026, 4:35 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 8, 2026, 4:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Identification of Genes Associated With Lung Disease in Patients With Rheumatoid Arthritis

NCT00001885
View directory record Official record
Conditions
Healthy, Pulmonary Fibrosis, Rheumatoid Arthritis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
21 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 8, 2026, 4:35 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 8, 2026, 4:35 PM 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 8, 2026, 4:35 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 Jul 16, 2026 · Synced Sep 8, 2026, 4:35 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 8, 2026, 4:35 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 8, 2026, 4:35 PM EDT
Terminated Phase 2 Interventional Results available

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

NCT04167345
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
VX-814, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
22
States / cities
Birmingham, Alabama • Sacramento, California • Denver, Colorado + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2022 · Synced Sep 8, 2026, 4:35 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 Sep 8, 2026, 4:35 PM EDT
Completed Phase 2Phase 3 Interventional

The Safety and Efficacy of Alpha-1 Antitrypsin (AAT) for the Prevention of Graft-versus-host Disease (GVHD) in Patients Receiving Hematopoietic Cell Transplant

NCT03805789
View directory record Official record
Conditions
Acute-graft-versus-host Disease
Interventions
AAT, Placebo
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
12 Years and older
Enrollment
222 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
12
States / cities
Scottsdale, Arizona • St. Petersburg, Florida • Atlanta, Georgia + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Sep 8, 2026, 4:35 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 8, 2026, 4:35 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 8, 2026, 4:35 PM EDT
Enrolling by invitation Phase 3 Interventional

Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency

NCT02796937
View directory record Official record
Conditions
Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
Interventions
Alpha-1 MP
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
20 Years to 72 Years
Enrollment
290 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Wilmington, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Sep 8, 2026, 4:35 PM 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 25, 2021 · Synced Sep 8, 2026, 4:35 PM EDT
Recruiting No phase listed Observational

PiMZ Longitudinal Cohort (PiMZ Logic)

NCT06505603
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema or COPD
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Sep 8, 2026, 4:35 PM EDT