Condition

Alpha 1-Antitrypsin Deficiency clinical trials and research

• 12Clinical trials
• 5NIH research projects
• 17Countries

Newest Alpha 1-Antitrypsin Deficiency clinical trials

All recruiting trials (12)

• Prediction score for death or lung transplant in emphysema caused by alpha-1 antitrypsin deficiency

  This study tests a new prediction tool to identify people with emphysema due to alpha-1-antitrypsin deficiency who are at high risk of death or needing a lung transplant.

  Recruiting at 4 sites University Hospital, Bordeaux · Bron and 3 other locations · NCT07715617

• Two weekly doses of Alpha1-Proteinase Inhibitor by injection compared with standard IV dose in alpha-1 antitrypsin deficiency

  A 'how it works' test to see if two weekly subcutaneous doses are as safe and help the body process the medicine as well as the standard IV dose for adults with AAT deficiency.

  Recruiting at 10 sites Phase 3 Grifols Therapeutics LLC · Birmingham, Alabama and 21 other locations · NCT07555483

• AIR-001 treatment in adults with alpha-1 antitrypsin deficiency

  This Phase 1 trial will test single ascending and multiple doses of AIR-001 for safety and how the drug is processed in adults with PiZZ alpha-1 antitrypsin deficiency.

  Recruiting at 11 sites Phase 1 AIRNA Corporation · Melbourne and 12 other locations · NCT07431112

• Gene therapy using AAV8 to replace defective alpha-1 antitrypsin

  This trial will try a one-time IV gene therapy (AAV8hAAT(AVL)) to produce an oxidation-resistant AAT protein in adults with ZZ or Z/null alpha-1 antitrypsin deficiency who have emphysema.

  Recruiting Phase 1 Weill Medical College of Cornell University · New York, New York · NCT06996756

• PiMZ Longitudinal Cohort: tracking lung changes in PiMZ Alpha-1 patients

  This project will test whether CT lung density and blood or sputum biomarkers can predict worsening emphysema over three years in adults with PiMZ alpha-1 antitrypsin deficiency and COPD.

  Recruiting Columbia University · Birmingham, Alabama and 4 other locations · NCT06505603

• Assessing a new treatment for Alpha-1 Antitrypsin Deficiency

  This study is testing a new treatment called WVE-006 to see if it is safe and effective for people with Alpha-1 Antitrypsin Deficiency who have mild to moderate lung or liver issues.

  Recruiting Phase 1/2 Wave Life Sciences USA, Inc. · Fitzroy, Victoria and 8 other locations · NCT06405633

• Evaluating BEAM-302 for treating Alpha-1 Antitrypsin Deficiency in adults

  This study is testing a new treatment called BEAM-302 to see if it can help adults with Alpha-1 Antitrypsin Deficiency who have lung or liver problems.

  Recruiting at 12 sites Phase 1/2 Beam Therapeutics Inc. · Birmingham, Alabama and 11 other locations · NCT06389877

• Treatment of Alpha-1 Antitrypsin Deficiency with KB408

  This study is testing a new treatment called KB408 to see if it can help adults with alpha-1 antitrypsin deficiency breathe better by delivering a key protein directly to their lungs.

  Recruiting at 3 sites Phase 1 Krystal Biotech, Inc. · Gainesville, Florida and 2 other locations · NCT06049082

• National registry for patients with alpha-1-antitrypsin deficiency

  This study collects health information from people with alpha-1-antitrypsin deficiency to better understand how the condition affects them over time.

  Recruiting Thomayer University Hospital · Prague, Czech Republic · NCT05178277

• Registry for individuals with Alpha-1 Antitrypsin Deficiency

  This study is creating a database to gather information from people with Alpha-1 Antitrypsin Deficiency and carriers to help researchers find better treatments and understand the disease better.

  Recruiting Alpha-1 Foundation · Coral Gables, Florida · NCT04157049

• Investigating how lung diseases affect white blood cells in the lungs

  This study is trying to see how Alpha-1-antitrypsin deficiency and Cystic Fibrosis affect important white blood cells in the lungs, which could help us understand lung inflammation and disease better.

  Recruiting University of Florida · Gainesville, Florida · NCT01851642

• Genetic testing for alpha-1 antitrypsin deficiency

  This study is testing how genetic testing can help people at risk for alpha-1 antitrypsin deficiency understand their health and any related conditions.

  Recruiting University of Florida · Gainesville, Florida · NCT00500123

NIH-funded research (5)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Understanding why AAT deficiency harms the liver and how to fix it

  A career development program will explore how a genetic liver disease (AAT deficiency) damages liver cells and test ideas that could lead to new treatments for people with ZZ mutations.

  NIH research Boston University Medical Campus · Boston, United States · NIH-11403995

• Gene therapy approach to treat Alpha-1 Antitrypsin Deficiency

  A gene therapy approach is being developed to treat Alpha-1 Antitrypsin deficiency, a lung disease, with the aim of providing a lasting source of the protective AAT protein.

  NIH research Weill Medical Coll of Cornell Univ · New York, United States · NIH-11370353

• Treating alpha-1 antitrypsin deficiency by targeting cellular protein-folding pathways

  This project uses small molecules to fix how cells fold alpha-1 antitrypsin so people with different genetic forms of alpha-1 antitrypsin deficiency may keep healthier livers and lungs.

  NIH research Scripps Research Institute, the · La Jolla, United States · NIH-11285254

• Managing Alpha-1-Antitrypsin Deficiency through Protein Pathways

  This study is looking at how to better treat Alpha-1-Antitrypsin Deficiency (AATD) by understanding how different gene variations affect the disease and testing new ways to help the body make the AAT protein work better, which could lead to improved treatments for patients like you.

  NIH research Scripps Research Institute, the · La Jolla, United States · NIH-11046628

• Creating a new tool to correct a genetic defect causing alpha-1 antitrypsin deficiency.

  This study is testing a new way to fix the genetic problem that causes alpha-1 antitrypsin deficiency by using a special tool to edit the gene in your liver, which could lead to better and safer treatments for people with this condition.

  NIH research Univ of Massachusetts Med Sch Worcester · Worcester, United States · NIH-10996880

Related topics

Quick answers

How many Alpha 1-Antitrypsin Deficiency clinical trials are recruiting right now?

12, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 17 countries. The most sites are in United States (8 trials), Australia (3 trials), Germany (3 trials), Netherlands (3 trials), New Zealand (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 3, Phase 1/2: 2, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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