Alpha-1-antitrypsin DeficiencyNCT05178277
Czech AATD Registry
An observational study of Alpha-1-antitrypsin Deficiency, run by Thomayer University Hospital at 1 site in Czechia.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alpha-1-antitrypsin Deficiency
- How many people
- 300 participants
- Where
- 1 siteCzechia
- Study dates
- 1 Jan 2018to 31 Dec 2035
- How long will I take part?
- 20 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with alpha-1-antitrypsin deficiency
Who may be able to join
- Patients with alpha-1-antitrypsin deficiency
Who cannot join
- Patient disagreement with inclusion in the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Czechia1 site, 1 recruiting
- Recruiting
Thomayer university hospital
Prague, Czech Republic, 14059
Jan Chlumsky, MD
About this study
Official title: Czech Alpha-1 Antitrypsin Deficiency Registry, the National Observational Study.
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.
Read the detailed description
Alpha-1 antitrypsin deficiency is a genetic disorder that may result in lung disease or liver disease. It is assume that it affects 1 person from a cohort of 2,000-5,000 people of the general population. Among patients with COPD, the incidence of the disorder is significantly higher. The prognosis of these patients is incomparably worse compared to classic COPD, because it affects younger patients and the rate of lung tissue loss is faster. The diagnosis is made in patients with pre-existing COPD by examination of the plasma concentration of AAT. In case of its reduction, genetic examination is added. The progression of the disease is rapid and has been shown to be slowed by lifelong augmentation treatment with human AAT. However, in routine clinical practice, it is very difficult to assess the effectiveness of treatment, the progression of lung disease or the prognosis of the disease.
The AATD registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The national registry collects data from all patients with severe or rare AAT deficiency, regardless of the type of organ impairment and age, and thus provides a view of this genetic variation in the Czech population.
The aim of the AATD Registry is to collect and analyse clinical data of patients with alpha-1 antitrypsin deficiency and increase the professional awareness of this hereditary disease.
- Conditions
- Alpha-1-antitrypsin Deficiency
- Keywords
- Alpha-1-antitrypsin deficiency, AATD, national registry
- Medical subject headings
- alpha 1-Antitrypsin Deficiency
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of lung function parameters over time
assessement of the rate of decline of FEV1 (ml, %predicted), measured annually
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: within one year after completion
Secondary outcomes (2)
Behavior of individuals with no or minimal lung involvement
Assessement of prognosis of deficient non-smokers by monitoring primary outcome parameters 1-3
Measured: within one year after completion
Progression of other organ disorders, namely liver
Laboratory detection of changes in liver tests (ALT, AST, ALP, GGT, ukat/l)
Measured: within one year after completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2022
- Study start1 Jan 2018
- Primary completion31 Dec 2033 (estimated)
- Study completion31 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alpha-1-antitrypsin DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 20 Years
Sponsor and investigators
- Lead sponsor
- Thomayer University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Masaryk University
- Responsible party
- Principal investigator, Jan Chlumsky, M.D., Thomayer University Hospital
- Investigators
- Jan MD Chlumsky, PhD, Principal investigator, Thomayer University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05178277
- Sponsor's protocol ID
- IBA1115
- Status last verified
- Dec 2021
- First submitted
- 12 Nov 2021
- Last updated
- 5 Jan 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Czech Alpha-1 Antitrypsin Deficiency Registry, the National Observational Study.
- ClinicalTrials.gov ID
- NCT05178277
- Acronym / sponsor ID
- IBA1115
- Conditions
- Alpha-1-antitrypsin Deficiency
- Keywords
- Alpha-1-antitrypsin deficiency, AATD, national registry
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Thomayer University HospitalUniversity, hospital or other
- Collaborators
- Masaryk University
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 31 Dec 2033 (estimated)
- Study completion
- 31 Dec 2035 (estimated)
- First posted
- 5 Jan 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Alpha-1 antitrypsin deficiency is a genetic disorder that may result in lung disease or liver disease. It is assume that it affects 1 person from a cohort of 2,000-5,000 people of the general population. Among patients with COPD, the incidence of the disorder is significantly higher. The prognosis of these patients is incomparably worse compared to classic COPD, because it affects younger patients and the rate of lung tissue loss is faster. The diagnosis is made in patients with pre-existing COPD by examination of the plasma concentration of AAT. In case of its reduction, genetic examination is added. The progression of the disease is rapid and has been shown to be slowed by lifelong augmentation treatment with human AAT. However, in routine clinical practice, it is very difficult to assess the effectiveness of treatment, the progression of lung disease or the prognosis of the disease.
The AATD registry is a non-interventional multicenter retrospective prospective longitudinal follow-up of patients with alpha-1-antitrypsin deficiency. The national registry collects data from all patients with severe or rare AAT deficiency, regardless of the type of organ impairment and age, and thus provides a view of this genetic variation in the Czech population.
The aim of the AATD Registry is to collect and analyse clinical data of patients with alpha-1 antitrypsin deficiency and increase the professional awareness of this hereditary disease.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 20 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with alpha-1-antitrypsin deficiency
Key inclusion criteria (1 listed)
- Patients with alpha-1-antitrypsin deficiency
Key exclusion criteria (1 listed)
- Patient disagreement with inclusion in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Changes of lung function parameters over time
Time frame: within one year after completion
assessement of the rate of decline of FEV1 (ml, %predicted), measured annually
Changes of exercise tolerance tolerance over time
Time frame: within one year after completion
assessment of changes of peak oxygen consumption (peakVO2, ml/kg/min) measured every two years
Changes of respiratory function over time
Time frame: within one year after completion
assessement of the rate of decline of TLco (mol/min/kPa, %predicted), measured annually
Changes of quality of life over time
Time frame: within one year after completion
assessement of the rate of decline of COPD assessment test (CAT, points), measured annually
Relationship of pulmonary function and lung CT densitometry to better determine phenotypes of COPD due to AAT deficiency
Time frame: within one year after completion
Assessement of any possible relationship of primary outcomes 1-3 using LAA (low attenuation area, %) and distribution of emphysema (craniocaudal distribution of emhysema, points)
2 secondary outcomes
Behavior of individuals with no or minimal lung involvement
Time frame: within one year after completion
Progression of other organ disorders, namely liver
Time frame: within one year after completion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jan MD Chlumsky, PhD principal investigator
Thomayer University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha-1-antitrypsin Deficiency, run by Thomayer University Hospital at 1 site in Czechia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Katerina Kusalova, Ing (+420723949465, kusalova@biostatistika.cz), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes of lung function parameters over time (time frame: within one year after completion). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2022.