Alpha 1-Antitrypsin Deficiency Clinical Trials

9 recruiting

Alpha 1-Antitrypsin Deficiency Trials at a Glance

10 actively recruiting trials for alpha 1-antitrypsin deficiency are listed on ClinicalTrialsFinder across 6 cities in 14 countries. The largest study group is Phase 1 with 4 trials, with the heaviest enrollment activity in Birmingham, New York, and Charleston. Lead sponsors running alpha 1-antitrypsin deficiency studies include Beam Therapeutics Inc., BioMarin Pharmaceutical, and Alpha-1 Foundation.

Browse alpha 1-antitrypsin deficiency trials by phase

About Alpha 1-Antitrypsin Deficiency Clinical Trials

Looking for clinical trials for Alpha 1-Antitrypsin Deficiency? There are currently 9 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 Deficiency 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 Deficiency 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 110 of 10 trials

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
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