Baby Observational and Nutritional Study

Cystic Fibrosis Clinical Trial

— BONUS  

Official title:

Baby Observational and Nutritional Study (BONUS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01424696
• Other study ID #: BONUS-IP-11
• Secondary ID: R01DK095738
• Status: Completed
• Phase: Phase 2
• First received: August 25, 2011
• Last updated: September 25, 2015
• Start date: December 2011
• Est. completion date: April 2015

Study information

• Verified date: September 2015
• Source: Seattle Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 231
• Est. completion date: April 2015
• Est. primary completion date: April 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 3 Months

Eligibility

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.

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency
• Failure to Thrive
• Growth Failure

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory CF Center	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Austin Children's Chest Associates	Austin	Texas
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	State University of New York at Buffalo	Buffalo	New York
United States	Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern Medical Center at Dallas	Dallas	Texas
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Helen DeVos Children's Hospital CF Care Center	Grand Rapids	Michigan
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Iowa City University of Iowa	Iowa City	Iowa
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Vanderbilt CF Center	Nashville	Tennessee
United States	University of Oklahoma Health Sciences Center	Oklahoma City	Oklahoma
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	University of Utah	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Cardinal Glennon Children's Medical Center	St. Louis	Missouri
United States	Washington University School of Medicine	St. Louis	Missouri
United States	SUNY Upstate Medical University	Syracuse	New York

Sponsors (3)

Lead Sponsor	Collaborator
Seattle Children's Hospital	Cystic Fibrosis Foundation Therapeutics, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incremental gain in weight, length, and head circumference	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	one year	No