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ClinicalTrials.gov public records Last synced Oct 30, 2026, 4:02 PM EDT

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Showing 1–24 of 84 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Alpha 1-Antitrypsin Deficiency Clear all
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 Oct 30, 2026, 4:02 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 14, 2025 · Synced Oct 30, 2026, 4:02 PM 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 12, 2021 · Synced Oct 30, 2026, 4:02 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 16, 2018 · Synced Oct 30, 2026, 4:02 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 15, 2024 · Synced Oct 30, 2026, 4:02 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 2, 2017 · Synced Oct 30, 2026, 4:02 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 23, 2019 · Synced Oct 30, 2026, 4:02 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 23, 2026 · Synced Oct 30, 2026, 4:02 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 13, 2026 · Synced Oct 30, 2026, 4:02 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 20, 2013 · Synced Oct 30, 2026, 4:02 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 13, 2018 · Synced Oct 30, 2026, 4:02 PM 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 Oct 30, 2026, 4:02 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 21, 2020 · Synced Oct 30, 2026, 4:02 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 28, 2019 · Synced Oct 30, 2026, 4:02 PM 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 21, 2023 · Synced Oct 30, 2026, 4:02 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 6, 2026 · Synced Oct 30, 2026, 4:02 PM EDT
Enrolling by invitation No phase listed Observational

AATD-Liver Scan Study

NCT07898553
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, AATD, Alpha-1 Protease Inhibitor Deficiency, Alpha-1 AT Deficiency, Alpha-1 Antitrypsin Deficiency (AATD)
Interventions
Not listed
Lead sponsor
Alpha-1 Foundation
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Coral Gables, Florida
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 30, 2026, 4:02 PM 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 24, 2026 · Synced Oct 30, 2026, 4:02 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 24, 2025 · Synced Oct 30, 2026, 4:02 PM EDT
Completed Early Phase 1 Interventional

Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency

NCT00377416
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV2-CB-hAAT Gene Vector
Genetic
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2020
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 10, 2020 · Synced Oct 30, 2026, 4:02 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 7, 2026 · Synced Oct 30, 2026, 4:02 PM 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 Oct 30, 2026, 4:02 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 17, 2021 · Synced Oct 30, 2026, 4:02 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 12, 2024 · Synced Oct 30, 2026, 4:02 PM EDT