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Baby Observational and Nutritional Study (BONUS)

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ClinicalTrials.gov Identifier: NCT01424696
Recruitment Status : Completed
First Posted : August 29, 2011
Last Update Posted : September 28, 2015
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Description
Brief Summary:

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

Detailed Description:

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 Design

Study Type : Observational
Actual Enrollment : 231 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Baby Observational and Nutritional Study (BONUS)
Study Start Date : December 2011
Primary Completion Date : April 2015
Study Completion Date : April 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

Groups and Cohorts

Group/Cohort
Cystic Fibrosis in 1st 3 months of life
Early Diagnosis: Children diagnosed with CF in first 3 months of life


Outcome Measures

Primary Outcome Measures :
  1. 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
  • urine
  • stool
  • oropharyngeal swab (OP swab)
  • saliva
  • sweat
  • buccal brushings

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 3 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Infants diagnosed with CF who attend regular clinic visits during the first year of life
Criteria

Observational Study

Inclusion Criteria:

  1. Signed informed consent
  2. Males or females no more than three and one half (3.5) months of age at enrollment
  3. Documentation of a CF diagnosis as evidenced by:

    1. One or more of the following: one or more clinical features consistent with the CF phenotype OR a positive newborn screening (NBS) OR a positive pre-natal screen

      AND

    2. One or more of the following: sweat chloride ≥ 60 mEq/liter by quantitative pilocarpine iontophoresis test (QPIT) OR two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
  4. Enrolled in the Cystic Fibrosis Foundation Patient Registry. (Patients may enroll in the Registry at Enrollment Visit if not previously enrolled.)

Exclusion Criteria:

  1. Children unable to take full oral feeds
  2. Any serious or active medical condition, which in the opinion of the investigator, contributes to malabsorption, interferes with normal growth, or would otherwise interfere with subject's treatment, assessment, or compliance with the protocol.
  3. Gestational age less than 35 weeks and/or birth weight < 2.5 kg.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01424696


  Show 28 Study Locations
Sponsors and Collaborators
Seattle Children's Hospital
Cystic Fibrosis Foundation Therapeutics
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
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
More Information

Responsible Party: Bonnie Ramsey, Professor, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT01424696     History of Changes
Other Study ID Numbers: BONUS-IP-11
R01DK095738 ( U.S. NIH Grant/Contract )
First Posted: August 29, 2011    Key Record Dates
Last Update Posted: September 28, 2015
Last Verified: September 2015

Keywords provided by Bonnie Ramsey, Seattle Children's Hospital:
growth measures
nutritional status
pulmonary infection
inflammatory markers
pancreatic enzyme replacement therapy

Additional relevant MeSH terms:
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Failure to Thrive
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Signs and Symptoms