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Benefits and Risks of Newborn Screening for Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00014950
Recruitment Status : Completed
First Posted : April 16, 2001
Last Update Posted : March 2, 2010
Sponsor:
Collaborator:
National Center for Research Resources (NCRR)
Information provided by:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Tracking Information
First Submitted Date  ICMJE April 14, 2001
First Posted Date  ICMJE April 16, 2001
Last Update Posted Date March 2, 2010
Study Start Date  ICMJE Not Provided
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00014950 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Benefits and Risks of Newborn Screening for Cystic Fibrosis
Official Title  ICMJE Pulmonary Benefits of Cystic Fibrosis Neonatal Screening
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 and/or 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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Single Group Assignment
Primary Purpose: Diagnostic
Condition  ICMJE
  • Cystic Fibrosis
  • Lung Disease
  • Pseudomonas Infections
Intervention  ICMJE Procedure: CF newborn screening
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE Not Provided
Original Enrollment  ICMJE Not Provided
Study Completion Date  ICMJE Not Provided
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Must have been born in the State of Wisconsin
  • Must have been born between April 15, 1985 and June 30, 1994
  • Must have had a valid newborn screening test for cystic fibrosis in the first 28 days of life.
  • Must have a sweat chloride test greater or equal to 60 mmol/Liter
  • Parental consent
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00014950
Other Study ID Numbers  ICMJE Farrell (completed)
R01DK034108 ( U.S. NIH Grant/Contract )
GCRC
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborators  ICMJE National Center for Research Resources (NCRR)
Investigators  ICMJE
Principal Investigator: Philip M. Farrell, MD, PhD Dean University of Wisconsin Medical School
Investigator: Michael J. Rock, M.D. Dept. Pediatrics, UW Hospital
Investigator: Mark Splaingard Children's Hospital and Health System Foundation, Wisconsin
Investigator: Anita Laxova Dept. Pediatrics, UW Madison
PRS Account National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Verification Date March 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP