Alpha-1 Antitrypsin Deficiency
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CLINICAL_TRIALS trial_result · 2026-04-17Clinical Trial Update: A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110 [Phase 1, Phase 2]
Korro Bio has terminated the Phase 1/2a study of KRRO-110 in Alpha-1 Antitrypsin Deficiency (AATD), which involved single- and multiple-dose…
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CLINICAL_TRIALS research · 2026-04-14Clinical Trial Update: A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD [Phase 1, Phase 2]
Phase 1b/2a open-label study in adults with Pi*ZZ Alpha-1 Antitrypsin Deficiency is active/not recruiting, sponsored by Wave Life Sciences.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema [Phase 2]
Open-label Phase 2 long-term study of SAR447537 (INBRX-101) in adults with Alpha-1 antitrypsin deficiency–emphysema; the trial is currently …
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD) [Phase 1]
AIR-001 is a Phase 1 study in adults with Alpha-1 Antitrypsin Deficiency and is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-23Clinical Trial Update: A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1) [Phase 1]
Phase 1 study assessing safety, tolerability, and pharmacokinetics of WVE-006 in healthy participants with wild-type alpha-1 antitrypsin exp…
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) [Phase 1, Phase 2]
This clinical trial update describes BEAM-302 being studied for safety and efficacy in adults with Alpha-1 antitrypsin deficiency in a Phase…
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: Gene Therapy for Alpha 1- Antitrypsin Deficiency [Phase 1]
Phase 1 clinical trial evaluating a gene therapy for Alpha-1 antitrypsin deficiency, currently recruiting.
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: 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) [Phase 4]
Phase 4, double-blind trial assessing CT lung density decline in adults with alpha-1 antitrypsin deficiency treated with Respreeza or Zemair…
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CLINICAL_TRIALS research · 2026-03-05Clinical Trial Update: 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 [Phase 3]
Phase 3 study to assess the safety and potential efficacy of Fazirsiran for liver disease and fibrosis caused by an abnormal alpha-1 antitry…
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CLINICAL_TRIALS research · 2026-03-05Clinical Trial Update: 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) [Phase 3]
A Phase 3 clinical trial is recruiting to evaluate the safety of Fazirsiran and its potential to help people with Alpha-1 Antitrypsin Defici…
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CLINICAL_TRIALS trial_result · 2026-03-04Clinical Trial Update: Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD) [Phase 3]
Phase 3 trial assessing the efficacy and safety of Alpha-1 MP (human alpha-1 proteinase inhibitor, modified process) in pulmonary emphysema …
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CLINICAL_TRIALS trial_result · 2026-03-04Clinical Trial Update: Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency [Phase 3]
This clinical trial update describes an ongoing Phase 3 study evaluating the long-term safety of alpha1-proteinase inhibitor in individuals …
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CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
This clinical trial update describes a longitudinal biomarker study cohort for Alpha-1 antitrypsin deficiency, sponsored by Columbia Univers…
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CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: PiMZ Longitudinal Cohort (PiMZ Logic)
The PiMZ Longitudinal Cohort trial sponsored by Columbia University is currently recruiting, focusing on individuals with Alpha-1 antitrypsi…
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Alpha-1 Foundation DNA and Tissue Bank
This clinical trial update notes that the Alpha-1 Foundation DNA and Tissue Bank study for Alpha-1 antitrypsin deficiency is enrolling by in…
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CLINICAL_TRIALS research · 2026-01-05Clinical Trial Update: Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer
This clinical trial update investigates how prevalent Alpha-1-antitrypsin deficiency is among patients with hepatocellular carcinoma arising…
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Study of ARO-AAT in Normal Adult Volunteers [Phase 1]
Phase 1 study of ARO-AAT in normal adult volunteers has completed. The trial relates to alpha-1 antitrypsin deficiency.
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CLINICAL_TRIALS trial_result · 2025-12-19Clinical Trial Update: Polygen Defi-Alpha: Genetic Polymorphisms Study in Children With Alpha-1 Antitrypsin Deficiency, Included in the DEFI-ALPHA Cohort [NA]
Completed genetic polymorphism study in children with Alpha-1 antitrypsin deficiency included in the DEFI-ALPHA cohort.
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CLINICAL_TRIALS withdrawal · 2025-12-18Clinical Trial Update: Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels [Phase 2]
Phase 2 study ARC-AAT for Alpha-1 Antitrypsin Deficiency was withdrawn; the trial evaluated safety, tolerability, and effects on circulating…
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CLINICAL_TRIALS trial_result · 2025-12-12Clinical Trial Update: 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 [Phase 1, Phase 2]
A completed Phase 1/2 trial evaluating the safety, tolerability and pharmacokinetics of two subcutaneous doses of Alpha1-Proteinase Inhibito…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study
The Alpha-1 Coded Testing (ACT) Study, a collaboration between the Alpha-1 Foundation and the University of Florida, is currently recruiting…
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CLINICAL_TRIALS trial_result · 2025-11-21Clinical Trial Update: Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency
A completed clinical trial evaluating the impact of pulmonary rehabilitation in patients with Alpha-1 antitrypsin deficiency.
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CLINICAL_TRIALS research · 2025-11-12Clinical Trial Update: A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD) [Phase 1, Phase 2]
A Phase 1/2 trial update indicates Tessera Therapeutics is evaluating TSRA-196 in adults with Alpha-1 Antitrypsin Deficiency (AATD); the stu…
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CLINICAL_TRIALS research · 2025-11-10Clinical Trial Update: Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency [Phase 4]
A Phase 4 clinical trial investigating the role of immunoglobulin G genetic variation in individuals with Alpha-1 antitrypsin deficiency and…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
University of Florida researchers are recruiting for a study on how lung disease affects the function of white blood cells in people with Al…