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ClinicalTrials.gov public records Last synced Oct 30, 2026, 4:03 PM EDT

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Showing 25–48 of 85 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Alpha-1 Protease Inhibitor Deficiency Clear all
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, 4:03 PM EDT
Withdrawn Phase 2 Interventional

A Research Trial of Aralast NP in New Onset Diabetes (RETAIN) - Part II

NCT01183455
View directory record Official record
Conditions
Diabetes Mellitus, Type 1
Interventions
Aralast NP, Placebo
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
15
States / cities
La Jolla, California • Aurora, Colorado • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 31, 2014 · Synced Oct 30, 2026, 4:03 PM EDT
Terminated Not applicable Interventional

Environment Effect on Six-Minute Walk Test Performance

NCT02502201
View directory record Official record
Conditions
Chronic Obstructive Pulmonary Disease, Alpha-1 Antitrypsin Deficiency
Interventions
six-minute walk study indoors, six-minute walk study outdoors
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Aug 22, 2019 · Synced Oct 30, 2026, 4:03 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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 Oct 8, 2026 · Synced Oct 30, 2026, 4:03 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

NCT01851642
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD, Cystic Fibrosis (CF)
Interventions
History and physical exam., Blood draw., Pulmonary function testing., Albuterol inhaler.
Procedure · Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2033
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 30, 2026, 4:03 PM EDT
Completed Phase 4 Interventional Results available

Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency

NCT00261833
View directory record Official record
Conditions
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Interventions
Alpha1-proteinase inhibitor, Placebo
Biological · Other
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 65 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
4
States / cities
Denver, Colorado • Miami, Florida • Hershey, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 19, 2015 · Synced Oct 30, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

Alpha-1 Research Registry

NCT04157049
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
Alpha-1 Foundation
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Coral Gables, Florida
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 30, 2026, 4:03 PM 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 12, 2016 · Synced Oct 30, 2026, 4:03 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 30, 2026, 4:03 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 30, 2026, 4:03 PM 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 4, 2025 · Synced Oct 30, 2026, 4:03 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 30, 2026, 4:03 PM EDT
Completed Phase 3 Interventional Results available

Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults

NCT00295061
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Alpha-1 MP, alpha-1 proteinase inhibitor (human)
Drug
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
18 Years and older
Enrollment
24 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 Sep 9, 2014 · Synced Oct 30, 2026, 4:03 PM EDT
Completed 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
2014 – 2026
U.S. locations
10
States / cities
Phoenix, Arizona • Gainesville, Florida • Leesburg, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 30, 2026, 4:03 PM EDT
Completed Phase 2 Interventional Results available

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

NCT03679598
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
Interventions
Alvelestat (MPH966), Placebo
Drug · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 80 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 30, 2026, 4:03 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 26, 2022 · Synced Oct 30, 2026, 4:03 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 30, 2026, 4:03 PM EDT
Terminated No phase listed Observational

Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency

NCT00005098
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 30, 2026, 4:03 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 2, 2022 · Synced Oct 30, 2026, 4:03 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 5, 2021 · Synced Oct 30, 2026, 4:03 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 30, 2026, 4:03 PM 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 13, 2026 · Synced Oct 30, 2026, 4:03 PM 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 8, 2016 · Synced Oct 30, 2026, 4:03 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 13, 2022 · Synced Oct 30, 2026, 4:03 PM EDT