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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
Terminated Phase 2 Interventional Results available

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

NCT02093221
View directory record Official record
Conditions
Type 1 Diabetes Mellitus
Interventions
180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI, Placebo
Biological
Lead sponsor
Grifols Therapeutics LLC
Industry
Eligibility
6 Years to 35 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
36
States / cities
Tucson, Arizona • Los Angeles, California • Orange, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2018 · Synced Jul 21, 2026, 11:53 AM 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 27, 2019 · Synced Jul 21, 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 Jul 21, 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 Jul 21, 2026, 11:53 AM 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 16, 2021 · Synced Jul 21, 2026, 11:53 AM 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 12, 2017 · Synced Jul 21, 2026, 11:53 AM 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 23, 2005 · Synced Jul 21, 2026, 11:53 AM 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 15, 2026 · Synced Jul 21, 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 – 2032
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Jul 21, 2026, 11:53 AM 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 20, 2020 · Synced Jul 21, 2026, 11:53 AM 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 4, 2021 · Synced Jul 21, 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 Jul 21, 2026, 11:53 AM EDT
Completed Phase 3 Interventional Results available

GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study

NCT02525861
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency
Interventions
GLASSIA
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
17
States / cities
Tucson, Arizona • Los Angeles, California • Sacramento, California + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2021 · Synced Jul 21, 2026, 11:53 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 Jul 21, 2026, 11:53 AM EDT
Completed Not applicable Interventional Results available

Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability

NCT01615484
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
Transplantation of lungs obtained from Non-Heart-Beating Donors (NHBDs) after ex-vivo perfusion w/ STEEN Solution™, STEEN Solution™
Procedure · Device
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
15 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
2
States / cities
Chapel Hill, North Carolina • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 7, 2018 · Synced Jul 21, 2026, 11:53 AM EDT
Recruiting Phase 3 Interventional

Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

NCT06165341
View directory record Official record
Conditions
Alpha1-Antitrypsin Deficiency
Interventions
Fazirsiran Injection, Placebo
Drug
Lead sponsor
Takeda
Industry
Eligibility
18 Years to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
20
States / cities
Phoenix, Arizona • Tucson, Arizona • La Jolla, California + 15 more
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Jul 21, 2026, 11:53 AM EDT
Active, not recruiting 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
2013 – 2027
U.S. locations
10
States / cities
Phoenix, Arizona • Gainesville, Florida • Leesburg, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Jul 21, 2026, 11:53 AM EDT
Completed Phase 1 Interventional

The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes

NCT01319331
View directory record Official record
Conditions
Diabetes, Type 1 Diabetes
Interventions
Alpha 1-Antitrypsin (AAT, Aralast NP)
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
6 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 23, 2017 · Synced Jul 21, 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 Jul 21, 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 Jul 21, 2026, 11:53 AM EDT
Completed Phase 2 Interventional

A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.

NCT03636347
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency, Emphysema, COPD
Interventions
Placebo Oral Tablet, Alvelestat oral tablet - dose 1, Alvelestat oral tablet - dose 2
Drug
Lead sponsor
Mereo BioPharma
Industry
Eligibility
18 Years to 75 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2022 · Synced Jul 21, 2026, 11:53 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 Jul 21, 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 Jul 21, 2026, 11:53 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 Jul 21, 2026, 11:53 AM EDT