Alpha1-antitrypsin Deficiency Registry

Related Clinical Trial
A Single Ascending and Repeated Dose Study of Oral ZF874 in Healthy Volunteers and PiMZ Subjects ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT) Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals COPD Exacerbation Blood and Urine Biomarkers Study Alpha-1 Carrier Genomics Study Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype Study of ARO-AAT in Normal Adult Volunteers Safety Study of Alfalastin (Human Alpha-1 Antitrypsin) Administered at Home AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry Respreeza® Self-administration and Learning Program (AmAREtTI Study) Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo. Environment Effect on Six-Minute Walk Test Performance Alpha-1 Foundation DNA and Tissue Bank Alpha1-antitrypsin Deficiency Registry Alpha-1 Research Registry Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency Alpha-1 Coded Testing(ACT) Study Long-Term Follow-up Study of ADVM-043 Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency 4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency? Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation” EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 MP in Patients With Alpha1-Antitrypsin Deficiency Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency Lung Volume Reduction Coils for Emphysema in Alpha-1 Antitrypsin Deficiency Study of Genotype and Phenotype in Patients With Alpha 1-Antitrypsin Deficiency Alpha-1 Foundation Research Registry Targeting Pulmonary Perfusion in Alpha-1 Antitrypsin Deficiency Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients Evaluate Efficacy and Safety of “Kamada-AAT for Inhalation” in Patients With AATD A Study of DCR-A1AT in Healthy Adult Volunteers and Patients With A1ATD-Associated Liver Disease Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency Effects of Different Exercise Training Modalities in Alpha-1 Antitrypsin Deficiency Patients Safety Study of an Aerosolized, Recombinant Alpha 1-Antitrypsin in Subjects With Alpha 1-Antitrypsin Deficiency Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE) Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 – Proteinase Inhibitor) Aralast alpha1-proteinase Inhibitor Surveillance Study Pharmacokinetic Study of ARALAST (Human Alpha1- PI) The Use of High Resolution Chest Computed Tomography in Alpha-1 Antitrypsin Deficiency Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD) Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency The Impact of Delayed Diagnosis of Alpha-1 Antitrypsin Deficiency Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD) A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency

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