Ignite Creation Date:
2024-05-05 @ 11:20 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00005292
Status:
COMPLETED
Last Update Posted:
2016-03-25
First Post:
2000-05-25
Brief Title:
Alpha1-antitrypsin Deficiency Registry
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2004-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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 Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: