search
Back to results

Alpha1-antitrypsin Deficiency Registry

Primary Purpose

Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency

Status
Completed
Phase
Locations
Study Type
Observational
Intervention
Sponsored by
National Heart, Lung, and Blood Institute (NHLBI)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an observational trial for Lung Diseases

Eligibility Criteria

undefined - 100 Years (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

No eligibility criteria

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    May 25, 2000
    Last Updated
    March 24, 2016
    Sponsor
    National Heart, Lung, and Blood Institute (NHLBI)
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT00005292
    Brief Title
    Alpha1-antitrypsin Deficiency Registry
    Study Type
    Observational

    2. Study Status

    Record Verification Date
    August 2004
    Overall Recruitment Status
    Completed
    Study Start Date
    September 1988 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    November 1999 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    National Heart, Lung, and Blood Institute (NHLBI)

    4. Oversight

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease

    7. Study Design

    10. Eligibility

    Sex
    Male
    Maximum Age & Unit of Time
    100 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    No eligibility criteria
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Mark Schluchter
    Organizational Affiliation
    The Cleveland Clinic

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    2331946
    Citation
    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.
    Results Reference
    background
    PubMed Identifier
    1480878
    Citation
    Schluchter 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.
    Results Reference
    background
    PubMed Identifier
    7924498
    Citation
    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.
    Results Reference
    background
    PubMed Identifier
    9106567
    Citation
    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. doi: 10.1378/chest.111.4.899.
    Results Reference
    background
    PubMed Identifier
    9041988
    Citation
    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. doi: 10.1378/chest.111.2.394.
    Results Reference
    background
    PubMed Identifier
    8970361
    Citation
    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. doi: 10.1164/ajrccm.154.6.8970361.
    Results Reference
    background
    PubMed Identifier
    10712324
    Citation
    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. doi: 10.1164/ajrccm.161.3.9906011.
    Results Reference
    background
    PubMed Identifier
    9655706
    Citation
    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. doi: 10.1164/ajrccm.158.1.9712017.
    Results Reference
    background
    PubMed Identifier
    12628876
    Citation
    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. doi: 10.1378/chest.123.3.765.
    Results Reference
    background
    Available IPD and Supporting Information:
    Available IPD/Information Type
    Individual Participant Data Set
    Available IPD/Information URL
    http://biolincc.nhlbi.nih.gov/studies/aadr
    Available IPD/Information Identifier
    AADR
    Available IPD/Information Comments
    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.
    Available IPD/Information Type
    Study Forms
    Available IPD/Information URL
    https://biolincc.nhlbi.nih.gov/studies/aadr/Forms/

    Learn more about this trial

    Alpha1-antitrypsin Deficiency Registry

    We'll reach out to this number within 24 hrs