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Alpha1-antitrypsin Deficiency Registry

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00005292
First Posted: May 26, 2000
Last Update Posted: March 25, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)
  Purpose
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.

Condition
Lung Diseases Emphysema Alpha-1 Antitrypsin Deficiency Chronic Obstructive Pulmonary Disease

Study Type: Observational

Resource links provided by NLM:


Further study details as provided by National Heart, Lung, and Blood Institute (NHLBI):

Study Start Date: September 1988
Estimated Study Completion Date: November 1999
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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 100 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria
No eligibility criteria
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005292


Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
OverallOfficial: Mark Schluchter The Cleveland Clinic
  More Information

Publications:
Stoller JK, Williams GW, Crystal RG. Alpha 1-antitrypsin deficiency registry. Chest. 1990 May;97(5):1278.
Schluchter MD. Methods for the analysis of informatively censored longitudinal data. Stat Med. 1992 Oct-Nov;11(14-15):1861-70.
A 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.
Stoller 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.
McElvaney 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.
Turino 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.
Schluchter 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.
Survival 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.
Eden 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.

Study Data/Documents: Individual Participant Data Set  This link exits the ClinicalTrials.gov site
Identifier: AADR
NHLBI provides controlled access to IPD through BioLINCC. Access requires registration, evidence of local IRB approval or certification of exemption from IRB review, and completion of a data use agreement.

ClinicalTrials.gov Identifier: NCT00005292     History of Changes
Other Study ID Numbers: 2014
First Submitted: May 25, 2000
First Posted: May 26, 2000
Last Update Posted: March 25, 2016
Last Verified: August 2004

Additional relevant MeSH terms:
Lung Diseases
Lung Diseases, Obstructive
Pulmonary Disease, Chronic Obstructive
Emphysema
Alpha 1-Antitrypsin Deficiency
Respiratory Tract Diseases
Pathologic Processes
Liver Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Alpha 1-Antitrypsin
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action


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