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ClinicalTrials.gov public records Last synced Oct 1, 2026, 2:49 PM EDT

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Showing 25–48 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Alpha1-proteinase Inhibitor Deficiency Clear all
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 Oct 1, 2026, 2:49 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 1, 2026, 2:49 PM 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 28, 2024 · Synced Oct 1, 2026, 2:49 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 1, 2026, 2:49 PM EDT
Terminated Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Safety and Efficacy of (α1Proteinase Inhibitor, α1PI) in HIV Disease

NCT01731691
View directory record Official record
Conditions
HIV Disease
Interventions
α1 Proteinase Inhibitor, Placebos
Biological · Drug
Lead sponsor
Institute for Human Genetics and Biochemistry
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 1, 2026, 2:49 PM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate the Safety and Efficacy of Liquid Alpha1-Proteinase Inhibitor (Human) in Hospitalized Participants With Coronavirus Disease (COVID-19)

NCT04547140
View directory record Official record
Conditions
COVID-19
Interventions
Liquid Alpha1-Proteinase Inhibitor (Human), Placebo, Standard Medical Treatment
Biological · Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
10
States / cities
Birmingham, Alabama • Phoenix, Arizona • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2023 · Synced Oct 1, 2026, 2:49 PM 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 Oct 1, 2026, 2:49 PM EDT
Completed Phase 1Phase 2 Interventional

Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency

NCT00157092
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
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 5, 2021 · Synced Oct 1, 2026, 2:49 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

NCT02282527
View directory record Official record
Conditions
Alpha₁-Antitrypsin Deficiency
Interventions
Liquid Alpha₁-PI, Prolastin-C
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years to 70 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
6
States / cities
Denver, Colorado • Gainesville, Florida • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2017 · Synced Oct 1, 2026, 2:49 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 20, 2024 · Synced Oct 1, 2026, 2:49 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 1, 2026, 2:49 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 1, 2026, 2:49 PM 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 3, 2014 · Synced Oct 1, 2026, 2:49 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 16, 2026 · Synced Oct 1, 2026, 2:49 PM 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 19, 2012 · Synced Oct 1, 2026, 2:49 PM 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 Oct 1, 2026, 2:49 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 1, 2026, 2:49 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 1, 2026, 2:49 PM 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 Oct 1, 2026, 2:49 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 1, 2026, 2:49 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 1, 2026, 2:49 PM 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 Oct 1, 2026, 2:49 PM 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 13, 2018 · Synced Oct 1, 2026, 2:49 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 22, 2019 · Synced Oct 1, 2026, 2:49 PM EDT