Baby Observational and Nutritional Study (BONUS)
|ClinicalTrials.gov Identifier: NCT01424696|
Recruitment Status : Completed
First Posted : August 29, 2011
Last Update Posted : September 28, 2015
Cystic fibrosis (CF) is a life-shortening disease that causes breathing and digestive problems, but can now be diagnosed at the time of birth. Lung function is very hard to measure in infants, but growth is not. In this study the investigators aim to define growth in infants with CF in the first year of life with research quality precision and to understand factors that interfere with good growth.
Pancreatic enzyme replacement therapy (PERT) will be also be studied in a subgroup of infants. Two different doses of PERT will be evaluated for improving fat and nitrogen absorption in infants with CF.
|Condition or disease||Phase|
|Cystic Fibrosis Growth Failure Exocrine Pancreatic Insufficiency||Phase 2|
Newborn screening (NBS) for cystic fibrosis (CF) has decreased the prevalence of malnutrition in infancy, but suboptimal nutrition still persists. In one study, 60% of infants diagnosed by NBS achieved their birth weight percentile by two years of age, while 40% did not. The many factors that contribute to this poor growth have not been defined and persist despite pancreatic enzyme supplementation. Although published guidelines for the clinical management of infants with cystic fibrosis in the U.S. and Europe exist, there is an alarming scarcity of evidence to dictate care. In order to proceed with large scale randomized studies to evaluate the range of interventions for infants with CF, we need to not only develop precise techniques for measuring growth but also pursue unexplored factors that may contribute to poor growth.
This is a multi-center observational clinical study with a nested interventional PERT sub-study. The observational study was designed to follow in a prospective manner incident cases of CF for up to 12 months. The PERT sub-study is a randomized, double-blind, crossover sub-study designed to evaluate the efficacy and safety of two doses of PERT (pancreatic enzyme replacement therapy) for improving coefficient of fat absorption (CFA) in the stool of infants with CF. The PERT sub-study was unable to enroll and closed. No results available.
|Study Type :||Observational|
|Actual Enrollment :||231 participants|
|Official Title:||Baby Observational and Nutritional Study (BONUS)|
|Study Start Date :||December 2011|
|Primary Completion Date :||April 2015|
|Study Completion Date :||April 2015|
Cystic Fibrosis in 1st 3 months of life
Early Diagnosis: Children diagnosed with CF in first 3 months of life
- Incremental gain in weight, length, and head circumference [ Time Frame: one year ]To define and describe incremental weight gain and linear growth in the first year of life utilizing research quality growth measures that will be applicable as efficacy outcomes for future interventional studies in infants with CF
Biospecimen Retention: Samples With DNA
- serum and/or plasma
- oropharyngeal swab (OP swab)
- buccal brushings
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01424696
Show 28 Study Locations
|Principal Investigator:||Drucy Borowitz, MD||State University of New York at Buffalo|
|Principal Investigator:||Daniel Leung, MD||Baylor College of Medicine|
|Principal Investigator:||James Heubi, MD||University of Cincinnati|
|Principal Investigator:||Daniel Gelfond, MD||Women & Children's Hospital of Buffalo|