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ClinicalTrials.gov public records Last synced Sep 14, 2026, 11:53 AM EDT

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Showing 1–24 of 83 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha 1 Antitrypsin Deficiency Clear all
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 14, 2026, 11:53 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 12, 2021 · Synced Sep 14, 2026, 11:53 AM 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 Sep 14, 2026, 11:53 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 14, 2026, 11:53 AM EDT
Completed No phase listed Observational

Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD)

NCT01419158
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
Interventions
Not listed
Lead sponsor
Alpha-1 Foundation
Other
Eligibility
18 Years and older
Enrollment
3,457 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
18
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2012 · Synced Sep 14, 2026, 11:53 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 25, 2021 · Synced Sep 14, 2026, 11:53 AM EDT
Terminated Phase 2 Interventional Results available

Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

NCT01379469
View directory record Official record
Conditions
Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
Interventions
Drug-Carbamazepine (Tegretol XR), Carbamazepine (Tegretol XR) Placebo
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
14 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced Sep 14, 2026, 11:53 AM 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 14, 2026, 11:53 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 17, 2020 · Synced Sep 14, 2026, 11:53 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 14, 2026, 11:53 AM 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 14, 2026, 11:53 AM 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 Sep 14, 2026, 11:53 AM 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 14, 2026, 11:53 AM EDT
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 23, 2026 · Synced Sep 14, 2026, 11:53 AM 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 Sep 14, 2026, 11:53 AM 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 14, 2026, 11:53 AM 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 Sep 14, 2026, 11:53 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 14, 2026, 11:53 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency

NCT02691611
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years to 85 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2020
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Sep 14, 2026, 11:53 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 14, 2026, 11:53 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 15, 2026 · Synced Sep 14, 2026, 11:53 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 12, 2026 · Synced Sep 14, 2026, 11:53 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 14, 2026, 11:53 AM 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 14, 2026, 11:53 AM EDT