Alpha1-antitrypsin Deficiency Registry

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

Study Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Study Sponsor

Mark Schluchter, , The Cleveland Clinic

Verification Date

August 2004