Alpha 1-Antitrypsin DeficiencyNCT00700934
Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema
An observational study of Alpha 1-Antitrypsin Deficiency and Emphysema, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The aim of this study is to describe the natural history of patients with alpha-1 antitrypsin associated emphysema and to figure out associated prognostic factors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency, Emphysema
- How many people
- 300 participants
- Where
- 1 siteFrance
- Study dates
- Feb 2005to Feb 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Emphysema associated alpha-1 antitrypsin deficiency
Who may be able to join
- Serum alpha one antitrypsin < 0.8 g/l
- Emphysema diagnosed by CT scan
- Adult
- Obstructive disorder
Who cannot join
- Liver or lung transplantation
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Departement d'épidémiologie, Hôpital Bichat
Paris, 75018
Gabriel Thabut, MD
About this study
Official title: Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema. A National Registry.
The aim of this study is to describe the natural history of patients with alpha-1 antitrypsin associated emphysema and to figure out associated prognostic factors.
Read the detailed description
All patients who suffer from this disease are followed every 6 months during a 5 years period. FEV1 decline is the primary endpoint. Quality of life assessed using the Saint George's Respiratory Questionary is recorded too.
- Conditions
- Alpha 1-Antitrypsin Deficiency, Emphysema
- Keywords
- FEV1, mortality, Quality of life
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Emphysema
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2008 (estimated)
- Study startFeb 2005
- Primary completionFeb 2011 (estimated)
- Study completionFeb 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Investigators
- Gabriel Thabut, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00700934
- Sponsor's protocol ID
- 001
- Status last verified
- Jun 2008
- First submitted
- 18 Jun 2008
- Last updated
- 7 Dec 2010
- Results posted
- No
- Data monitoring committee
- No
Publications
Gauvain C, Mornex JF, Pison C, Cuvelier A, Balduyck M, Pujazon MC, Fournier M, AitIlalne B, Thabut G. Health-related quality of life in patients with alpha-1 antitrypsin deficiency: the French experience. COPD. 2015 May;12 Suppl 1:46-51. doi: 10.3109/15412555.2015.1022645.
PubMed 25938292Thabut G, Mornex JF, Pison C, Cuvelier A, Balduyck M, Pujazon MC, Fournier M, AitIlalne B, Porcher R. Performance of the BODE index in patients with alpha1-antitrypsin deficiency-related COPD. Eur Respir J. 2014 Jul;44(1):78-86. doi: 10.1183/09031936.00168113. Epub 2014 Feb 13.
PubMed 24525449
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema. A National Registry.
- ClinicalTrials.gov ID
- NCT00700934
- Acronym / sponsor ID
- 001
- Conditions
- Alpha 1-Antitrypsin Deficiency, Emphysema
- Keywords
- FEV1, mortality, Quality of life
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Feb 2005
- Primary completion
- Feb 2011 (estimated)
- Study completion
- Feb 2012 (estimated)
- First posted
- 19 Jun 2008 (estimated)
- 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.
The aim of this study is to describe the natural history of patients with alpha-1 antitrypsin associated emphysema and to figure out associated prognostic factors.
Detailed description
All patients who suffer from this disease are followed every 6 months during a 5 years period. FEV1 decline is the primary endpoint. Quality of life assessed using the Saint George's Respiratory Questionary is recorded too.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Emphysema associated alpha-1 antitrypsin deficiency
Key inclusion criteria (4 listed)
- Serum alpha one antitrypsin < 0.8 g/l
- Emphysema diagnosed by CT scan
- Adult
- Obstructive disorder
Key exclusion criteria (1 listed)
- Liver or lung transplantation
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
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2010.
- Derived from this study: PubMed 25938292Gauvain C, Mornex JF, Pison C, Cuvelier A, Balduyck M, Pujazon MC, Fournier M, AitIlalne B, Thabut G. Health-related quality of life in patients with alpha-1 antitrypsin deficiency: the French experience. COPD. 2015 May;12 Suppl 1:46-51. doi: 10.3109/15412555.2015.1022645.
- Derived from this study: PubMed 24525449Thabut G, Mornex JF, Pison C, Cuvelier A, Balduyck M, Pujazon MC, Fournier M, AitIlalne B, Porcher R. Performance of the BODE index in patients with alpha1-antitrypsin deficiency-related COPD. Eur Respir J. 2014 Jul;44(1):78-86. doi: 10.1183/09031936.00168113. Epub 2014 Feb 13.
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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. This study is not recruiting at the moment.
Gabriel Thabut, MD principal investigator
Assistance Publique - Hôpitaux de Paris
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 and Emphysema, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 4 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: status not confirmed, as of the sponsor's last update on 7 Dec 2010. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Gabriel Thabut, MD (1 40 87 57 12, g.thabut@bch.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00700934, which also lists 2 linked publications.
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 7 Dec 2010.