Lung DiseasesNCT00005292
Alpha1-antitrypsin Deficiency Registry
An observational study of Lung Diseases and Emphysema, run by National Heart, Lung, and Blood Institute (NHLBI).
Trial summary
To collect data from the 37 participating clinical centers on patients with alpha1-antitrypsin deficiency, including those who received replacement therapy with an intravenous preparation of alpha1-proteinase inhibitor (A1Pi) concentrate. Read more about this study
- Who is it for?
- Ages Up to 100 years, men only
- Conditions the study is for
- Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Sep 1988to Nov 1999
- 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 Up to 100 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
No eligibility criteria
About this study
To collect data from the 37 participating clinical centers on patients with alpha1-antitrypsin deficiency, including those who received replacement therapy with an intravenous preparation of alpha1-proteinase inhibitor (A1Pi) concentrate.
Read the detailed description
BACKGROUND:
Severe congenital deficiency for alpha1-antitrypsin is associated with the early onset of emphysema, usually by the third decade of life. One approach to correct this deficiency is though replacement with alpha1-antitrypsin (referred to as alpha1-proteinase (A1Pi) inhibitor in its purified form). An intravenous preparation of A1Pi concentrate was produced from human plasma by Cutter Biological, a division of Miles, Inc., Berkeley, California. This preparation had been evaluated in a clinical study for its safety and biochemical efficacy. Based on the augmentation of its levels in the lung upon intravenous administration, the A1Pi preparation was licensed by the Food and Drug Administration for replacement therapy to treat individuals with severe congenital deficiency and impaired lung function. When the registry began in 1988, clinical efficacy was plausible, but unproven and there was no data base for estimating the degree of clinical benefit, if any.
Slow progression of emphysema and lack of an adequate control group have made it difficult to evaluate the proteinase inhibitor through a controlled clinical trial. A patient registry was an alternative method to collect data on the effect of long-term replacement therapy with A1Pi on rate of decline of lung function. The registry also included individuals who did not receive the replacement therapy in order to obtain a better knowledge of the rate of decline of lung function associated with the congenital deficiency for alpha1-antitrypsin.
DESIGN NARRATIVE:
The registry consisted of a clinical coordinating center, 37 participating clinical centers that contributed patient data to the registry, a steering committee, and a data analysis and policy board, both appointed by the National Heart, Lung, and Blood Institute. Data collected on all patients included a clinical history, laboratory evaluations such as chest x-ray, lung function studies of vital capacity, total lung capacity, forced expiratory volume in one second (FEV1) and blood studies. In addition, patients receiving replacement therapy had baseline lung function tests, spirometry every six months following initiation of replacement therapy, and measurements of serum alpha1-antitrypsin level pre- and post-infusion, once every six months. The recruitment phase ended in September 1990. Support for the registry ended in June, 1998.
- Conditions
- Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
- Medical subject headings
- Lung Diseases, Emphysema, alpha 1-Antitrypsin Deficiency, Pulmonary Disease, Chronic Obstructive
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2000 (estimated)
- Study startSep 1988
- Study completionNov 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Lung DiseasesStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- Mark Schluchter, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00005292
- Sponsor's protocol ID
- 2014
- Status last verified
- Aug 2004
- First submitted
- 25 May 2000
- Last updated
- 25 Mar 2016
- Results posted
- No
Publications
Stoller JK, Williams GW, Crystal RG. Alpha 1-antitrypsin deficiency registry. Chest. 1990 May;97(5):1278. doi: 10.1378/chest.97.5.1278b. No abstract available.
PubMed 2331946Schluchter MD. Methods for the analysis of informatively censored longitudinal data. Stat Med. 1992 Oct-Nov;11(14-15):1861-70. doi: 10.1002/sim.4780111408.
PubMed 1480878A registry of patients with severe deficiency of alpha 1-antitrypsin. Design and methods. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest. 1994 Oct;106(4):1223-32.
PubMed 7924498Stoller JK, Buist AS, Burrows B, Crystal RG, Fallat RJ, McCarthy K, Schluchter MD, Soskel NT, Zhang R. Quality control of spirometry testing in the registry for patients with severe alpha1-antitrypsin deficiency. alpha1-Antitrypsin Deficiency Registry Study Group. Chest. 1997 Apr;111(4):899-909. doi: 10.1378/chest.111.4.899.
PubMed 9106567McElvaney NG, Stoller JK, Buist AS, Prakash UB, Brantly ML, Schluchter MD, Crystal RD. Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest. 1997 Feb;111(2):394-403. doi: 10.1378/chest.111.2.394.
PubMed 9041988Turino GM, Barker AF, Brantly ML, Cohen AB, Connelly RP, Crystal RG, Eden E, Schluchter MD, Stoller JK. Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1996 Dec;154(6 Pt 1):1718-25. doi: 10.1164/ajrccm.154.6.8970361.
PubMed 8970361Schluchter MD, Stoller JK, Barker AF, Buist AS, Crystal RG, Donohue JF, Fallat RJ, Turino GM, Vreim CE, Wu MC. Feasibility of a clinical trial of augmentation therapy for alpha(1)-antitrypsin deficiency. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 2000 Mar;161(3 Pt 1):796-801. doi: 10.1164/ajrccm.161.3.9906011.
PubMed 10712324Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1998 Jul;158(1):49-59. doi: 10.1164/ajrccm.158.1.9712017.
PubMed 9655706Eden E, Hammel J, Rouhani FN, Brantly ML, Barker AF, Buist AS, Fallat RJ, Stoller JK, Crystal RG, Turino GM. Asthma features in severe alpha1-antitrypsin deficiency: experience of the National Heart, Lung, and Blood Institute Registry. Chest. 2003 Mar;123(3):765-71. doi: 10.1378/chest.123.3.765.
PubMed 12628876
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alpha1-antitrypsin Deficiency Registry
- ClinicalTrials.gov ID
- NCT00005292
- Acronym / sponsor ID
- 2014
- Conditions
- Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
- Study type
- Observational
- Recruitment status
- Completed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Sep 1988
- Study completion
- Nov 1999
- First posted
- 26 May 2000 (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.
To collect data from the 37 participating clinical centers on patients with alpha1-antitrypsin deficiency, including those who received replacement therapy with an intravenous preparation of alpha1-proteinase inhibitor (A1Pi) concentrate.
Detailed description
BACKGROUND:
Severe congenital deficiency for alpha1-antitrypsin is associated with the early onset of emphysema, usually by the third decade of life. One approach to correct this deficiency is though replacement with alpha1-antitrypsin (referred to as alpha1-proteinase (A1Pi) inhibitor in its purified form). An intravenous preparation of A1Pi concentrate was produced from human plasma by Cutter Biological, a division of Miles, Inc., Berkeley, California. This preparation had been evaluated in a clinical study for its safety and biochemical efficacy. Based on the augmentation of its levels in the lung upon intravenous administration, the A1Pi preparation was licensed by the Food and Drug Administration for replacement therapy to treat individuals with severe congenital deficiency and impaired lung function. When the registry began in 1988, clinical efficacy was plausible, but unproven and there was no data base for estimating the degree of clinical benefit, if any.
Slow progression of emphysema and lack of an adequate control group have made it difficult to evaluate the proteinase inhibitor through a controlled clinical trial. A patient registry was an alternative method to collect data on the effect of long-term replacement therapy with A1Pi on rate of decline of lung function. The registry also included individuals who did not receive the replacement therapy in order to obtain a better knowledge of the rate of decline of lung function associated with the congenital deficiency for alpha1-antitrypsin.
DESIGN NARRATIVE:
The registry consisted of a clinical coordinating center, 37 participating clinical centers that contributed patient data to the registry, a steering committee, and a data analysis and policy board, both appointed by the National Heart, Lung, and Blood Institute. Data collected on all patients included a clinical history, laboratory evaluations such as chest x-ray, lung function studies of vital capacity, total lung capacity, forced expiratory volume in one second (FEV1) and blood studies. In addition, patients receiving replacement therapy had baseline lung function tests, spirometry every six months following initiation of replacement therapy, and measurements of serum alpha1-antitrypsin level pre- and post-infusion, once every six months. The recruitment phase ended in September 1990. Support for the registry ended in June, 1998.
Study design
Who may qualify
- Ages
- Up to 100 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2016.
- Background: PubMed 2331946Stoller JK, Williams GW, Crystal RG. Alpha 1-antitrypsin deficiency registry. Chest. 1990 May;97(5):1278. doi: 10.1378/chest.97.5.1278b. No abstract available.
- Background: PubMed 1480878Schluchter MD. Methods for the analysis of informatively censored longitudinal data. Stat Med. 1992 Oct-Nov;11(14-15):1861-70. doi: 10.1002/sim.4780111408.
- Background: PubMed 7924498A registry of patients with severe deficiency of alpha 1-antitrypsin. Design and methods. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest. 1994 Oct;106(4):1223-32.
- Background: PubMed 9106567Stoller JK, Buist AS, Burrows B, Crystal RG, Fallat RJ, McCarthy K, Schluchter MD, Soskel NT, Zhang R. Quality control of spirometry testing in the registry for patients with severe alpha1-antitrypsin deficiency. alpha1-Antitrypsin Deficiency Registry Study Group. Chest. 1997 Apr;111(4):899-909. doi: 10.1378/chest.111.4.899.
- Background: PubMed 9041988McElvaney NG, Stoller JK, Buist AS, Prakash UB, Brantly ML, Schluchter MD, Crystal RD. Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group. Chest. 1997 Feb;111(2):394-403. doi: 10.1378/chest.111.2.394.
- Background: PubMed 8970361Turino GM, Barker AF, Brantly ML, Cohen AB, Connelly RP, Crystal RG, Eden E, Schluchter MD, Stoller JK. Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1996 Dec;154(6 Pt 1):1718-25. doi: 10.1164/ajrccm.154.6.8970361.
- Background: PubMed 10712324Schluchter MD, Stoller JK, Barker AF, Buist AS, Crystal RG, Donohue JF, Fallat RJ, Turino GM, Vreim CE, Wu MC. Feasibility of a clinical trial of augmentation therapy for alpha(1)-antitrypsin deficiency. The Alpha 1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 2000 Mar;161(3 Pt 1):796-801. doi: 10.1164/ajrccm.161.3.9906011.
- Background: PubMed 9655706Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1998 Jul;158(1):49-59. doi: 10.1164/ajrccm.158.1.9712017.
- Background: PubMed 12628876Eden E, Hammel J, Rouhani FN, Brantly ML, Barker AF, Buist AS, Fallat RJ, Stoller JK, Crystal RG, Turino GM. Asthma features in severe alpha1-antitrypsin deficiency: experience of the National Heart, Lung, and Blood Institute Registry. Chest. 2003 Mar;123(3):765-71. doi: 10.1378/chest.123.3.765.
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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.
Mark Schluchter
The Cleveland Clinic
Questions about this trial
What is this clinical trial studying?
An observational study of Lung Diseases and Emphysema, run by National Heart, Lung, and Blood Institute (NHLBI).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: finished, as of the sponsor's last update on 25 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00005292, which also lists 9 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 25 Mar 2016.