Cystic Fibrosis Clinical Trial
Official title:
Baby Observational and Nutritional Study (BONUS)
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.
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.
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Observational Model: Cohort, Time Perspective: Prospective
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