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ClinicalTrials.gov 9 recruiting now official registry

Alpha-1 Antitrypsin Deficiency clinical trials

9 of 19 indexed Alpha-1 Antitrypsin Deficiency trials are currently recruiting.

19 US clinical trials · 9 currently recruiting

Alpha-1 Antitrypsin Deficiency is the subject of 19 registered US clinical trials on ClinicalTrials.gov, 9 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 8 in Phase 1-2 (earlier-stage). The most active sponsor is Columbia University, running 2 of these trials.

The research picture

Alpha-1 Antitrypsin Deficiency has 19 registered US clinical trials, 9 of them open to new participants right now, about 47% of the total.

9
recruiting participants now
47%
of trials open to enrollment
1
in Phase 3–4 (later-stage)
2
top sponsor: Columbia University
Phase 15Phase 23Phase 31

Active & Recent Trials

19 total, page 1 of 1

RECRUITING 50,000 participants

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

University of Florida

NCT00500123

RECRUITING 4,000 participants

Alpha-1 Research Registry

Alpha-1 Foundation

NCT04157049

RECRUITING 220 participants

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

University of Florida

NCT01851642

RECRUITING Phase 2 185 participants

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

Sanofi

NCT05897424

RECRUITING Phase 1 106 participants

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

Beam Therapeutics

NCT06389877

RECRUITING 80 participants

PiMZ Longitudinal Cohort (PiMZ Logic)

Columbia University

NCT06505603

RECRUITING Phase 1 16 participants

Gene Therapy for Alpha 1- Antitrypsin Deficiency

Weill Medical College of Cornell University

NCT06996756

RECRUITING Phase 1 15 participants

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

Krystal Biotech

NCT06049082

RECRUITING Phase 1 12 participants

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

BioMarin Pharmaceutical

NCT06738017

ACTIVE NOT RECRUITING 286 participants

Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

Columbia University

NCT05297812

ACTIVE NOT RECRUITING 120 participants

Alpha-1 Antitrypsin Deficiency Adult Liver Study

St. Louis University

NCT02014415

COMPLETED 117 participants

Alpha-1 Carrier Genomics Study

Medical University of South Carolina

NCT02810327

COMPLETED Phase 1 114 participants

A Phase 1, First-in-human Study of VX-668

Vertex Pharmaceuticals Incorporated

NCT05727800

COMPLETED 43 participants

The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency

Biocerna

NCT03039335

COMPLETED Phase 3 38 participants

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Grifols Therapeutics

NCT00301366

COMPLETED NA 24 participants

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

University of North Carolina, Chapel Hill

NCT01615484

COMPLETED Phase 2 10 participants

Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.

Michael Campos, MD

NCT01669421

COMPLETED Phase 2 9 participants

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

Beacon Therapeutics

NCT01054339

COMPLETED 6 participants

Long-Term Follow-up Study of ADVM-043

Adverum Biotechnologies

NCT03804021

Phase Distribution

PhaseTrial count
Phase 1 5
Phase 2 3
Phase 3 1

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Alpha-1 Antitrypsin Deficiency Trial Landscape

19 indexed Alpha-1 Antitrypsin Deficiency studies · 9 recruiting (47%).

Phase mix: 1 late (III/IV) vs 8 early (I/II).

Top sponsor: Columbia University (2 trials), 9 others listed. 19 shown below, recruiting first.

How to read this condition's trial landscape

That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Alpha-1 Antitrypsin Deficiency?

PlainTrial tracks 19 US clinical trials for Alpha-1 Antitrypsin Deficiency, of which 9 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Alpha-1 Antitrypsin Deficiency?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

Verify with NIH →

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.