Brief Title
Alpha1-antitrypsin Deficiency Registry
Brief 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.
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 Type
Observational
Condition
Lung Diseases
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Start Date
September 1988
Completion Date
November 1999
Eligibility Criteria
No eligibility criteria
Gender
Male
Ages
N/A - 100 Years
Accepts Healthy Volunteers
No
Contacts
Mark Schluchter, ,
Administrative Informations
NCT ID
NCT00005292
Organization ID
2014
Study Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Study Sponsor
Mark Schluchter, , The Cleveland Clinic
Verification Date
August 2004