Alpha 1-Antitrypsin Clinical Trials

17 recruitingLast updated: June 17, 2026

There are 17 actively recruiting alpha 1-antitrypsin clinical trials across 17 countries. Studies span Phase 1, Phase 2, Phase 3, Not Applicable, Phase 4. Top locations include Birmingham, Alabama, United States, Gainesville, Florida, United States, Charleston, South Carolina, United States. Updated daily from ClinicalTrials.gov.


Alpha 1-Antitrypsin Trials at a Glance

17 actively recruiting trials for alpha 1-antitrypsin are listed on ClinicalTrialsFinder across 6 cities in 17 countries. The largest study group is Phase 1 with 6 trials, with the heaviest enrollment activity in Birmingham, Gainesville, and Charleston. Lead sponsors running alpha 1-antitrypsin studies include University of Florida, Beam Therapeutics Inc., and Alpha-1 Foundation.

Browse alpha 1-antitrypsin trials by phase

Treatments under study

About Alpha 1-Antitrypsin Clinical Trials

Looking for clinical trials for Alpha 1-Antitrypsin? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Alpha 1-Antitrypsin trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Alpha 1-Antitrypsin clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 117 of 17 trials

Recruiting
Phase 3

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)

Alpha 1-Antitrypsin Deficiency
Grifols Therapeutics LLC40 enrolled19 locationsNCT07555483
Recruiting
Phase 1Phase 2

A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-antitrypsin Deficiency (AATD)
Tessera Therapeutics, Inc.72 enrolled3 locationsNCT07227207
Recruiting
Phase 1

A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-Antitrypsin Deficiency
AIRNA Corporation54 enrolled4 locationsNCT07431112
Recruiting
Phase 1Phase 2

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-Antitrypsin Deficiency
Beam Therapeutics Inc.106 enrolled11 locationsNCT06389877
Recruiting
Phase 1

Gene Therapy for Alpha 1- Antitrypsin Deficiency

Alpha 1-Antitrypsin Deficiency
Weill Medical College of Cornell University16 enrolled1 locationNCT06996756
Recruiting

PiMZ Longitudinal Cohort (PiMZ Logic)

Alpha 1-Antitrypsin DeficiencyEmphysema or COPD
Columbia University80 enrolled5 locationsNCT06505603
Recruiting

The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study

Alpha 1-Antitrypsin Deficiency
University of Florida50,000 enrolled1 locationNCT00500123
Recruiting

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

Alpha 1-Antitrypsin DeficiencyCystic Fibrosis (CF)AAT Deficiency+1 more
University of Florida220 enrolled1 locationNCT01851642
Recruiting
Phase 4

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

COPDAlpha 1-AntitrypsinAntibody Deficiency
University of Alabama at Birmingham30 enrolled1 locationNCT07135427
Recruiting
Phase 2

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

EmphysemaAlpha 1-Antitrypsin Deficiency
Sanofi185 enrolled40 locationsNCT05897424
Recruiting

Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction

Alpha 1-antitrypsin Deficiency (AATD)
Muğla Sıtkı Koçman University734 enrolled1 locationNCT07152834
Recruiting
Phase 1

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Alpha 1-Antitrypsin Deficiency
BioMarin Pharmaceutical12 enrolled8 locationsNCT06738017
Recruiting
Phase 3

Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"

Alpha 1-Antitrypsin Deficiency
Kamada, Ltd.220 enrolled9 locationsNCT04204252
Recruiting
Phase 1

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

Alpha 1-Antitrypsin Deficiency
Krystal Biotech, Inc.15 enrolled3 locationsNCT06049082
Recruiting
Not Applicable

Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease

AsthmaChronic Obstructive Pulmonary DiseasePulmonary Fibrosis+5 more
McMaster University60 enrolled1 locationNCT03455686
Recruiting

Alpha-1 Research Registry

Alpha 1-Antitrypsin Deficiency
Alpha-1 Foundation4,000 enrolled1 locationNCT04157049
Recruiting

Czech AATD Registry

Alpha 1-Antitrypsin Deficiency
Thomayer University Hospital300 enrolled1 locationNCT05178277