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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Alpha 1 Antitrypsin Deficiency Clear all
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 May 31, 2026 · Synced Jun 26, 2026, 9:49 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 14, 2016 · Synced Jun 26, 2026, 9:49 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 18, 2014 · Synced Jun 26, 2026, 9:49 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 Jun 26, 2026, 9:49 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

NCT03945292
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Fazisiran Injection, Placebo
Drug · Other
Lead sponsor
Arrowhead Pharmaceuticals
Industry
Eligibility
18 Years to 75 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
16
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 AM 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 Jun 15, 2026 · Synced Jun 26, 2026, 9:49 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 Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 AM EDT
Completed Phase 2 Interventional

Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema

NCT05856331
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema
Interventions
SAR447537, Zemaira
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2025 · Synced Jun 26, 2026, 9:49 AM 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 19, 2013 · Synced Jun 26, 2026, 9:49 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 7, 2019 · Synced Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 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 25, 2022 · Synced Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

GLASSIA Infusion Rate Study

NCT01651351
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency, Healthy Volunteers
Interventions
Alpha1-proteinase inhibitor, Placebo: Human albumin 2.5%
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated May 24, 2021 · Synced Jun 26, 2026, 9:49 AM EDT
Completed No phase listed Observational

FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)

NCT02922751
View directory record Official record
Conditions
Biliary Atresia, Alagille Syndrome, Alpha1 Anti-Trypsin Deficiency, Portal Hypertension, Liver Fibrosis, Cholestasis
Interventions
Liver Stiffness Measurement (LSM)
Other
Lead sponsor
Arbor Research Collaborative for Health
Other
Eligibility
Up to 21 Years
Enrollment
552 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2024 · Synced Jun 26, 2026, 9:49 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 Jun 26, 2026, 9:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals

NCT00001462
View directory record Official record
Conditions
Emphysema, Lung Diseases, Obstructive, Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jun 26, 2026, 9:49 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 5, 2026 · Synced Jun 26, 2026, 9:49 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 14, 2026 · Synced Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 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 Jun 26, 2026, 9:49 AM 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 Jun 26, 2026, 9:49 AM EDT