Alpha-1 Antitrypsin Deficiency
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CLINICAL_TRIALS research · 2025-10-24Clinical Trial Update: A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
Takeda is conducting a clinical study in adults to learn about inherited Alpha-1 antitrypsin deficiency (AATD) and AATD-related liver proble…
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Update indicating the LOGIC genetic study of intrahepatic cholestasis is currently suspended.
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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 research · 2025-09-26Clinical Trial Update: Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) [EARLY_Phase 1]
A Phase 1 trial to study YOLT-202 for Alpha-1 antitrypsin deficiency is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction
A Muğla Sıtkı Koçman University trial is currently recruiting to screen patients with airway obstruction for Alpha-1 antitrypsin deficiency.
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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 research · 2025-09-08Clinical Trial Update: Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants [Phase 1]
A Phase 1 single-dose study of BMN 349 in adults with Alpha-1 antitrypsin deficiency is currently recruiting.
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CLINICAL_TRIALS research · 2025-09-04Clinical Trial Update: Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation" [Phase 3]
This update reports a Phase III trial of Kamada-AAT for inhalation for Alpha-1 antitrypsin deficiency, evaluating efficacy and safety; the s…
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CLINICAL_TRIALS research · 2025-08-19Clinical Trial Update: Alpha-1 Antitrypsin Deficiency Adult Liver Study
This clinical trial update indicates that the Alpha-1 Antitrypsin Deficiency adult liver study sponsored by St. Louis University is currentl…
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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 research · 2025-07-22Clinical Trial Update: A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency [Phase 1]
Phase 1 clinical trial update for KB408 in Alpha-1 Antitrypsin Deficiency, with study currently recruiting.
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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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EMA approval · 2025-03-27EMA Authorised: Respreeza (human alpha1-proteinase inhibitor) — CSL Behring GmbH
The EMA authorised Respreeza (human alpha1-proteinase inhibitor) for alpha-1 antitrypsin deficiency–related lung disease.
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CLINICAL_TRIALS research · 2025-03-24Clinical Trial Update: Preparation of IPSC for Cell Gene Editing for the Treatment of AATD [NA]
This clinical trial update describes preparing induced pluripotent stem cells for gene editing to treat Alpha-1 antitrypsin deficiency, with…
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CLINICAL_TRIALS withdrawal · 2025-01-17Clinical Trial Update: NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease [Phase 1, Phase 2]
A Phase 1/2 trial of NTLA-3001 for Alpha-1 Antitrypsin Deficiency–associated lung disease has been withdrawn.
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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 withdrawal · 2024-09-19Clinical Trial Update: An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) [Phase 2]
The Phase 2 extension study of belcesiran for Alpha-1 Antitrypsin Deficiency-associated liver disease has been withdrawn. Sponsor: Dicerna P…
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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…