Alpha-1 Antitrypsin Deficiency
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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 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 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 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 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 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…
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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 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 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 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 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 research · 2024-05-21Clinical Trial Update: Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease [NA]
Clinical trial update describing a study on hyperpolarized 129Xe MRI to image lung function in healthy volunteers and patients with various …