Czech AATD Registry

NCT05178277 | Recruiting

• 1 other identifier: IBA1115
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

Brief Summary

Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking. Patients with alpha-1-antitrypsin deficiency may also have liver cirrhosis, vasculitis, skin or intestinal disorders. The AATD Registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The aim of the AATD National Registry is to collect and analyze clinical data in patients with alpha-1 antitrypsin deficiency.

Conditions

Alpha-1-antitrypsin Deficiency

Keywords

Alpha-1-antitrypsin deficiency; AATD; national registry

Outcome Measures

Primary Outcomes (5)

• Changes of lung function parameters over time

  assessement of the rate of decline of FEV1 (ml, %predicted), measured annually

  within one year after completion

• Changes of exercise tolerance tolerance over time

  assessment of changes of peak oxygen consumption (peakVO2, ml/kg/min) measured every two years

  within one year after completion

• Changes of respiratory function over time

  assessement of the rate of decline of TLco (mol/min/kPa, %predicted), measured annually

  within one year after completion

• Changes of quality of life over time

  assessement of the rate of decline of COPD assessment test (CAT, points), measured annually

  within one year after completion

• Relationship of pulmonary function and lung CT densitometry to better determine phenotypes of COPD due to AAT deficiency

  Assessement of any possible relationship of primary outcomes 1-3 using LAA (low attenuation area, %) and distribution of emphysema (craniocaudal distribution of emhysema, points)

  within one year after completion

Secondary Outcomes (2)

• Behavior of individuals with no or minimal lung involvement

  within one year after completion

• Progression of other organ disorders, namely liver

  within one year after completion

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients with alpha-1-antitrypsin deficiency

You may qualify if:

• Patients with alpha-1-antitrypsin deficiency

Sponsors & Collaborators

• Thomayer University Hospital: lead
• Masaryk University: collaborator

Study Sites (1)

Thomayer university hospital

Prague, Czech Republic, 14059, Czechia

RECRUITING

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jan MD Chlumsky, PhD

  Thomayer University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Katerina Kusalova, Ing

CONTACT

+420723949465; kusalova@biostatistika.cz

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 20 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 12, 2021
• First Posted: January 5, 2022
• Study Start: January 1, 2018
• Primary Completion (Estimated): December 31, 2033
• Study Completion (Estimated): December 31, 2035
• Last Updated: January 5, 2022

Record last verified: 2021-12

Locations

CZ (1)