alpha 1-Antitrypsin Deficiency clinical trials
The registry lists 142 studies for alpha 1-Antitrypsin Deficiency across more than 10 countries. 32 are recruiting or about to start. 109 of them are mainly about alpha 1-Antitrypsin Deficiency (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
Grifols Therapeutics LLC (12) · Baxalta now part of Shire (8) · University of Florida (6) · Takeda (5) · Vertex Pharmaceuticals Incorporated (5) · Arrowhead Pharmaceuticals (5) · CSL Behring (4) · Kamada, Ltd. (4)
What is being tested here
Drugs under test in alpha 1-Antitrypsin Deficiency studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
9studies finish main data collection within a year1 of them phase 3
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 9, phase 3 first
- Sep 2027A 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) · Phase 3 · Grifols Therapeutics LLC
- Dec 2026Permian Health Women's Lung Health Study · Permian Health Lung Institute
- Jan 2027Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease · Columbia University
- Jan 2027Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) · RenJi Hospital
- Mar 2027Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency · University of Alabama at Birmingham
- Aug 2027Chronic Respiratory Disease Participant Engagement Study · Richmond Research Institute
- Sep 2027Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer · University Hospital, Bordeaux
- Sep 2027A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD · Wave Life Sciences USA, Inc.
- Sep 2027Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease. · Assiut University
Who these studies take
Of the 23 alpha 1-Antitrypsin Deficiency studies recruiting now, 11 (48%) set no upper age limit and 9 (39%) stop at 75 or younger. 4 (17%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 5 (22%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 4 (17%) | 4 (17%) |
| 18 | 22 (96%) | 22 (96%) |
| 30 | 23 (100%) | 23 (100%) |
| 40 | 23 (100%) | 23 (100%) |
| 50 | 23 (100%) | 23 (100%) |
| 65 | 23 (100%) | 23 (100%) |
| 75 | 17 (74%) | 17 (74%) |
| 85 | 13 (57%) | 13 (57%) |
Across all 142 studies, recruiting or not: 37% stop at 75 or younger and 15% at 65; 15% take children; 1% are for women only and 1% for men only; 20% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Started recruiting3
- IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel SyndromeNov 10, 2026NCT07707635
- A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATDOct 19, 2026NCT06405633
- Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)Oct 16, 2026NCT07326592
Newly registered3
- Chronic Respiratory Disease Participant Engagement StudyNov 9, 2026NCT07924462
- AATD-Liver Scan StudyOct 27, 2026NCT07898553
- A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin DeficiencyOct 21, 2026NCT07887347
Studies
Recruiting first, then most recently updated. Showing 50 of 142. Browse all 142 → or search them.
- 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)RecruitingNCT06165341Phase 313 countries
- A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)RecruitingNCT07227207Phase 1/25 countries
- IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel SyndromeRecruitingNCT07707635Not applicable1 country
- Chronic Respiratory Disease Participant Engagement StudyRecruitingNCT079244621 country
- Role of Genetic Factors in the Development of Lung DiseaseRecruitingNCT000015321 country
- AATD-Liver Scan StudyEnrolling by invitationNCT078985531 country
- Genetic Variation in IgG in Alpha 1 Antitrypsin DeficiencyRecruitingNCT07135427Phase 41 country
- 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)RecruitingNCT07555483Phase 39 countries
- A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)RecruitingNCT07431112Phase 19 countries
- A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin DeficiencyNot yet recruitingNCT07887347Phase 1/2
- A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATDRecruitingNCT06405633Phase 1/26 countries
- Gene Therapy for Alpha 1- Antitrypsin DeficiencyRecruitingNCT06996756Phase 11 country
- Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)RecruitingNCT07326592Phase 41 country
- A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)RecruitingNCT06389877Phase 1/26 countries
- A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)Not yet recruitingNCT07850076Phase 1/2
- Alpha-1 Research RegistryRecruitingNCT041570491 country
- A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver ProblemsRecruitingNCT065124546 countries
- Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin DeficiencyRecruitingNCT077156171 country
- Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin DeficiencyEnrolling by invitationNCT02796937Phase 312 countries
- Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.Not yet recruitingNCT07639996
- 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 ProteinRecruitingNCT05677971Phase 319 countries
- PiMZ Longitudinal Cohort (PiMZ Logic)RecruitingNCT065056031 country
- Alpha-1 Foundation DNA and Tissue BankEnrolling by invitationNCT008844551 country
- Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver CancerNot yet recruitingNCT071453851 country
- The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) StudyRecruitingNCT005001231 country
- Lung Disease and Its Affect on the Work of White Blood Cells in the LungsRecruitingNCT018516421 country
- Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway ObstructionRecruitingNCT071528341 country
- A Study of KB408 for the Treatment of Alpha-1 Antitrypsin DeficiencyRecruitingNCT06049082Phase 11 country
- Preparation of IPSC for Cell Gene Editing for the Treatment of AATDEnrolling by invitationNCT06892236Not applicable1 country
- Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung DiseaseRecruitingNCT03455686Not applicable1 country
- Permian Health Women's Lung Health StudyNot yet recruitingNCT060295431 country
- Czech AATD RegistryRecruitingNCT051782771 country
- An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver DiseaseActive, not recruitingNCT05899673Phase 35 countries
- A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin DeficiencyCompletedNCT04722887Phase 1/21 country
- Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)CompletedNCT01983241Phase 317 countries
- Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"TerminatedNCT04204252Phase 36 countries
- Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)SuspendedNCT005712722 countries
- Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult ParticipantsActive, not recruitingNCT06738017Phase 12 countries
- Alpha-1 Antitrypsin Deficiency Adult Liver StudyActive, not recruitingNCT020144151 country
- A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110TerminatedNCT06677307Phase 1/22 countries
- Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency EmphysemaActive, not recruitingNCT05897424Phase 29 countries
- A Study of Belcesiran in Patients With AATLDTerminatedNCT04764448Phase 214 countries
- A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)CompletedNCT06186492Phase 11 country
- Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of DiseaseActive, not recruitingNCT052978121 country
- A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)TerminatedNCT02363946Phase 14 countries
- Study of ARO-AAT in Normal Adult VolunteersCompletedNCT03362242Phase 11 country
- Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin LevelsWithdrawnNCT02900183Phase 24 countries
- Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin DeficiencyCompletedNCT072420791 country
- Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)CompletedNCT03945292Phase 26 countries
- Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)CompletedNCT03946449Phase 23 countries