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 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 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 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 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 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 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 trial_result · 2025-10-14Clinical Trial Update: An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease [Phase 3]
An extension phase 3 trial update to evaluate the long-term safety of Fazirsiran and its potential ongoing benefit for people with Alpha-1 a…
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CLINICAL_TRIALS trial_result · 2025-09-08Clinical Trial Update: A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype [Phase 2]
A Phase 2 study of VX-864 in Alpha-1 Antitrypsin Deficiency (PiZZ genotype) was terminated, and results have been posted.
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CLINICAL_TRIALS trial_result · 2025-08-11Clinical Trial Update: Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema [Phase 2]
Phase 2 study evaluating SAR447537 (INBRX-101) vs plasma-derived A1PI therapy in adults with Alpha 1-Antitrypsin Deficiency–related emphysem…
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CLINICAL_TRIALS trial_result · 2025-06-06Clinical Trial Update: Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
A completed clinical trial investigating epigenetic regulation of immunity in Alpha-1 antitrypsin deficiency.
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CLINICAL_TRIALS trial_result · 2025-05-28Clinical Trial Update: Respreeza® Self-administration and Learning Program (AmAREtTI Study)
The AmAREtTI study evaluating Respreeza self-administration and a learning program for Alpha-1 Antitrypsin Deficiency has completed.
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CLINICAL_TRIALS trial_result · 2025-04-20Clinical Trial Update: Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) [Phase 2]
Phase 2 study of Fazirsiran (TAK-999, ARO-AAT) for Alpha-1 antitrypsin deficiency–associated liver disease has completed with results posted…
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CLINICAL_TRIALS trial_result · 2024-12-31Clinical Trial Update: A Study of Belcesiran in Patients With AATLD [Phase 2]
A Phase 2 study of Belcesiran in patients with Alpha-1 antitrypsin deficiency has been terminated, with results posted.
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CLINICAL_TRIALS trial_result · 2024-12-30Clinical Trial Update: Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) [Phase 2]
Phase 2 trial evaluating Fazirsiran (TAK-999, ARO-AAT) in alpha-1 antitrypsin deficiency assessed safety, tolerability and pharmacodynamic e…
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CLINICAL_TRIALS trial_result · 2024-11-06Clinical Trial Update: Study of DCR-A1AT in Healthy Adult Volunteers [Phase 1]
Phase 1 trial of DCR-A1AT in healthy adult volunteers has completed.
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CLINICAL_TRIALS trial_result · 2024-09-20Clinical Trial Update: Long-Term Follow-up Study of ADVM-043
Long-term follow-up study of ADVM-043 in Alpha-1 antitrypsin deficiency has completed, with results posted in 2024.
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CLINICAL_TRIALS trial_result · 2024-08-28Clinical Trial Update: FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE)
This update reports on the FORCE trial evaluating FibroScan, a non-invasive liver fibrosis assessment tool, in pediatric cholestatic liver d…
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CLINICAL_TRIALS trial_result · 2024-07-09Clinical Trial Update: Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency [Phase 2]
Phase 2 trial of Alvelestat (MPH966) for Alpha-1 antitrypsin deficiency has completed with results posted.
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CLINICAL_TRIALS trial_result · 2024-06-04Clinical Trial Update: COPD Exacerbation Blood and Urine Biomarkers Study
A clinical trial evaluating blood and urine biomarkers in chronic obstructive pulmonary disease, including acute exacerbations and alpha-1 a…
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CLINICAL_TRIALS trial_result · 2022-08-08Clinical Trial Update: Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency [Phase 1, Phase 2]
Phase 1/2 safety and dose-finding study of ADVM-043 gene therapy for Alpha-1 Antitrypsin Deficiency has completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-04-14Clinical Trial Update: A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. [Phase 2]
A Phase 2 trial evaluating alvelestat (MPH966) versus placebo in participants with alpha1-antitrypsin-related COPD has completed.
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CLINICAL_TRIALS trial_result · 2022-02-02Clinical Trial Update: Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype [Phase 2]
Phase 2 trial evaluating VX-814 for Alpha 1-Antitrypsin Deficiency (PiZZ genotype) was terminated; results posted in 2022.