National registry for patients with alpha-1-antitrypsin deficiency

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The AATD Registry is a non-interventional multicenter observational study that collects and analyzes clinical data from patients diagnosed with alpha-1-antitrypsin deficiency, a genetic disorder that can lead to severe lung and liver diseases. This registry aims to provide a comprehensive view of the condition's impact on the Czech population by following patients longitudinally over time. It includes individuals with varying degrees of organ impairment and seeks to enhance awareness and understanding of this hereditary disease among healthcare professionals.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Patients with alpha-1-antitrypsin deficiency

Exclusion Criteria:

* Patient disagreement with inclusion in the study

Where it's running

• Thomayer university hospital
  Prague, Czech Republic, Czechia Recruiting

Study contacts

• Principal investigator: Jan MD Chlumsky, PhD — Thomayer University Hospital
• Study coordinator: Katerina Kusalova, Ing
• Email: kusalova@biostatistika.cz
• Phone: +420723949465

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• 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

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.