Ignite Creation Date:
2024-05-05 @ 11:23 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00014950
Status:
COMPLETED
Last Update Posted:
2010-03-02
First Post:
2001-04-14
Brief Title:
Benefits and Risks of Newborn Screening for Cystic Fibrosis
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Study Overview
Official Title:
Pulmonary Benefits of Cystic Fibrosis Neonatal Screening
Status:
COMPLETED
Status Verified Date:
2010-03
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:
Although cystic fibrosis CF is the most common life-threatening autosomal recessive genetic disorder of the white population there are often delays in diagnosis and hence start of treatment Advances of the past two decades have made CF screening feasible using routinely collected neonatal blood specimens and measuring an enzyme level followed by CF mutation DNA analysis Our overall goal of the study is to see if early diagnosis of CF through neonatal screening will be medically beneficial without major risks Medically beneficial refers to better nutrition andor pulmonary status whereas risks include laboratory errors miscommunication or misunderstanding and adverse psychosocial consequences Specific aims include assessment of the benefits risks costs quality of life and cognitive function associated with CF neonatal screening and a better understanding of the epidemiology of CF
A comprehensive randomized clinical trial emphasizing early diagnosis as the key variable has been underway since 1985 Nutritional status has been assessed using height and weight measurements and biochemical methods The results have demonstrated significant benefits in the screened early diagnosis group We are now focusing on the effect of early diagnosis of CF on pulmonary outcome Pulmonary status is measured using chest radiographs chest scans using high resolution computerized tomography and pulmonary function tests Other factors that we are looking at include risk factors for the acquisition of respiratory pathogens such as Pseudomonas aeruginosa quality of life and cognitive function of children with CF who underwent early versus delayed diagnosis as well as the cost effectiveness of screening and the costs of diagnosis and treatment of CF throughout childhood
If the questions underlying this study are answered favorably it is likely that neonatal screening using a combination of enzyme level immunoreactive trypsinogen and DNA test will become the routine method for identifying new cases of CF not only in the State of Wisconsin but throughout the country
A comprehensive randomized clinical trial emphasizing early diagnosis as the key variable has been underway since 1985 Nutritional status has been assessed using height and weight measurements and biochemical methods The results have demonstrated significant benefits in the screened early diagnosis group We are now focusing on the effect of early diagnosis of CF on pulmonary outcome Pulmonary status is measured using chest radiographs chest scans using high resolution computerized tomography and pulmonary function tests Other factors that we are looking at include risk factors for the acquisition of respiratory pathogens such as Pseudomonas aeruginosa quality of life and cognitive function of children with CF who underwent early versus delayed diagnosis as well as the cost effectiveness of screening and the costs of diagnosis and treatment of CF throughout childhood
If the questions underlying this study are answered favorably it is likely that neonatal screening using a combination of enzyme level immunoreactive trypsinogen and DNA test will become the routine method for identifying new cases of CF not only in the State of Wisconsin but throughout the country
Detailed Description:
None
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?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| GCRC | US NIH GrantContract | None | httpsreporternihgovquickSearchR01DK034108 |
| R01DK034108 | NIH | None | None |