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Alpha-1 Antitrypsin Deficiency: early-phase trial registry
12 of 25 indexed Alpha-1 Antitrypsin Deficiency trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #652 of 28,707 conditions by registered trial count.
25 US clinical trials · 12 currently recruiting · #652 of 28,707 conditions by registered trial count
Alpha-1 Antitrypsin Deficiency: 25 US clinical trials tracked, 12 recruiting.
Alpha-1 Antitrypsin Deficiency is the subject of 25 registered US clinical trials on ClinicalTrials.gov, 12 of them currently open to new participants. 4 are in Phase 3-4 (later-stage) and 10 in Phase 1-2 (earlier-stage). The most active sponsor is Takeda, running 4 of these trials.
Enrollment posture brief
Registry condition alpha-1-antitrypsin-deficiency
OPEN 12 | RECR 48% | LATE 4 | EARLY 10 | RANK #652 | SPON 4
Volume-matched condition peers
Nearest open-share peer: Diabetes Type 2 (48% recruiting · 25 trials)
Alpha-1 Antitrypsin Deficiency lists 12 open slots (48% of 25 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Alpha-1 Antitrypsin Deficiency on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Alpha-1 Antitrypsin Deficiency is recruiting above its size-band peers.
12 of 25 indexed trials (48%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Alpha-1 Antitrypsin Deficiency research.
Takeda accounts for 4 of 25 trials (16%). 18 distinct sponsors appear on at least one study for this condition label.
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Typical Alpha-1 Antitrypsin Deficiency enrolment sits around 50 participants.
Median target enrolment is 50 among the 25 trials that report a genuine target (sentinel values excluded). 14 of those (56%) plan for fewer than 100 participants.
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Alpha-1 Antitrypsin Deficiency splits between interventional and observational designs.
15 interventional and 10 observational studies make up this index (60% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
25 total, page 1 of 1
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study
University of Florida
NCT00500123
Alpha-1 Research Registry
Alpha-1 Foundation
NCT04157049
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Takeda
NCT06512454
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
University of Florida
NCT01851642
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Sanofi
NCT05897424
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
Takeda
NCT05677971
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Beam Therapeutics
NCT06389877
PiMZ Longitudinal Cohort (PiMZ Logic)
Columbia University
NCT06505603
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)
Takeda
NCT06165341
Gene Therapy for Alpha 1- Antitrypsin Deficiency
Weill Medical College of Cornell University
NCT06996756
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
Krystal Biotech
NCT06049082
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
BioMarin Pharmaceutical
NCT06738017
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Columbia University
NCT05297812
Alpha-1 Antitrypsin Deficiency Adult Liver Study
St. Louis University
NCT02014415
An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
Takeda
NCT05899673
Alpha-1 Carrier Genomics Study
Medical University of South Carolina
NCT02810327
A Phase 1, First-in-human Study of VX-668
Vertex Pharmaceuticals Incorporated
NCT05727800
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Vertex Pharmaceuticals Incorporated
NCT04474197
The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency
Biocerna
NCT03039335
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
Grifols Therapeutics
NCT00301366
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability
University of North Carolina, Chapel Hill
NCT01615484
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
Grifols Therapeutics
NCT04722887
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
Michael Campos, MD
NCT01669421
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Beacon Therapeutics
NCT01054339
Long-Term Follow-up Study of ADVM-043
Adverum Biotechnologies
NCT03804021
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 6 |
| Phase 2 | 4 |
| Phase 3 | 4 |
Top Sponsors
Named interventions
Most-linked intervention names on Alpha-1 Antitrypsin Deficiency records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Alpha-1 Antitrypsin Deficiency trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Alpha-1 Antitrypsin Deficiency page
25 registered trials, 12 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 12 Alpha-1 Antitrypsin Deficiency trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Takeda sponsors the most Alpha-1 Antitrypsin Deficiency trials on record, review their full trial history before evaluating a specific study. See Takeda's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Alpha-1 Antitrypsin Deficiency condition card totals 25 registered trials; most-linked intervention Placebo; top US site state Florida. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.