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Alpha1-Antitrypsin Deficiency
Open-data reference.
6 US clinical trials · 3 currently recruiting
Active & Recent Trials
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Takeda
NCT06512454
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
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
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
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Vertex Pharmaceuticals Incorporated
NCT04474197
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
Phase Distribution
| Phase | Trial count |
|---|---|
| Early Phase 1 | 1 |
| Phase 2 | 1 |
| Phase 3 | 3 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Alpha1-Antitrypsin Deficiency Trial Landscape
ClinicalTrials.gov lists 6 US studies indexed under Alpha1-Antitrypsin Deficiency, and 3 of those are currently open to recruitment — roughly 50% of the total volume on the registry. 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.
The phase distribution for Alpha1-Antitrypsin Deficiency shows 3 late-stage studies (Phase 3 and Phase 4 combined) alongside 2 earlier-phase entries (Phase 1 through Phase 2). 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.
Top sponsor activity for Alpha1-Antitrypsin Deficiency is led by Takeda with 4 indexed trials, alongside 2 other organizations in the top contributor list. The list on this page surfaces up to 6 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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 Alpha1-Antitrypsin Deficiency?
PlainTrial tracks 6 US clinical trials for Alpha1-Antitrypsin Deficiency, of which 3 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Alpha1-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.
Related
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.
Read our methodology — how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.