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Home › Conditions › Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Alpha-1 Antitrypsin Deficiency

16 trials tracked for Alpha-1 Antitrypsin Deficiency: 3 in phase 3 or 4.

16Trials tracked
3Phase 3 & 4
0Recruiting
0With published results
Phase distribution
Phase 4 1 Phase 3 2 Phase 2 8 Phase 1 2 Other / NA 3
  1. Phase 4 Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor) Completed
  2. Phase 3 Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Completed
  3. Phase 3 The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Completed
  4. Phase 2 Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation. Completed
  5. Phase 2 Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation" Completed
  6. Phase 2 Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) Completed
Show 10 more trials
  1. Phase 2 Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Completed
  2. Phase 2 Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE) Completed
  3. Phase 2 Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Completed
  4. Phase 2 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency Completed
  5. Phase 2 Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) Completed
  6. Phase 1 Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Completed
  7. Phase 1 Pharmacokinetic Study of ARALAST (Human Alpha1- PI) Completed
  8. N/A Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability Completed
  9. N/A Alpha-1 Carrier Genomics Study Completed
  10. N/A Long-Term Follow-up Study of ADVM-043 Completed

Showing the 16 most-cited and recently-updated of 16 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Heterozygous SERPINA1 genotypes are associated with increased liver disease risks in Alpha-1 Antitrypsin Deficiency
Gastroenterology Meta-analysis
Heterozygous SERPINA1 genotypes are associated with increased liver disease risks in Alpha-1 Antitrypsin Deficiency Specific gene variants linked to increased liver disease risks
This meta-analysis examined the impact of heterozygous SERPINA1 genotypes on various liver outcomes. The authors found that while enzyme lev…
People with certain genetic markers show significantly higher risks of liver scarring and failure, even without high body weight or fat leve…
Aug 1, 2026
Systematic review shows high rates of fibrosis and cirrhosis in children with Pi*ZZ alpha-1 antitrypsin deficiency
Gastroenterology Meta-analysis
Systematic review shows high rates of fibrosis and cirrhosis in children with Pi*ZZ alpha-1 antitrypsin deficiency 1 in 4 children with A1AT deficiency develop cirrhosis
This systematic review and meta-analysis examined children with confirmed Pi*ZZ alpha-1 antitrypsin deficiency to assess liver outcomes.
New analysis finds 41% of children with A1AT deficiency have liver fibrosis and 17% have cirrhosis.
May 1, 2026