Baby Observational and Nutritional Study

NCT ID: NCT01424696

Last Updated: 2015-09-28

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 231 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2011-12-31
• Study Completion Date: 2015-04-30

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.

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.

Conditions

Cystic Fibrosis; Growth Failure; Exocrine Pancreatic Insufficiency

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Cystic Fibrosis in 1st 3 months of life

Early Diagnosis: Children diagnosed with CF in first 3 months of life

No interventions assigned to this group

Eligibility Criteria

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.

• Maximum Eligible Age: 3 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role: collaborator

Seattle Children's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Bonnie Ramsey

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Drucy Borowitz, MD

Role: PRINCIPAL_INVESTIGATOR

State University of New York at Buffalo

Daniel Leung, MD

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

James Heubi, MD

Role: PRINCIPAL_INVESTIGATOR

University of Cincinnati

Daniel Gelfond, MD

Role: PRINCIPAL_INVESTIGATOR

Women & Children's Hospital of Buffalo

Locations

University of Alabama at Birmingham

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Emory CF Center

Atlanta, Georgia, United States

Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Iowa City University of Iowa

Iowa City, Iowa, United States

University of Michigan

Ann Arbor, Michigan, United States

Helen DeVos Children's Hospital CF Care Center

Grand Rapids, Michigan, United States

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

Cardinal Glennon Children's Medical Center

St Louis, Missouri, United States

Washington University School of Medicine

St Louis, Missouri, United States

State University of New York at Buffalo

Buffalo, New York, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Oregon Health and Science University

Portland, Oregon, United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Vanderbilt CF Center

Nashville, Tennessee, United States

Austin Children's Chest Associates

Austin, Texas, United States

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

Texas Children's Hospital

Houston, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Seattle Children's Hospital

Seattle, Washington, United States

Countries

United States

References

Sathe M, Huang R, Heltshe S, Eng A, Borenstein E, Miller SI, Hoffman L, Gelfond D, Leung DH, Borowitz D, Ramsey B, Freeman AJ. Gastrointestinal Factors Associated With Hospitalization in Infants With Cystic Fibrosis: Results From the Baby Observational and Nutrition Study. J Pediatr Gastroenterol Nutr. 2021 Sep 1;73(3):395-402. doi: 10.1097/MPG.0000000000003173.

Reference Type: DERIVED

PMID: 34016873 (View on PubMed)

Goetz D, Kopp BT, Salvator A, Moore-Clingenpeel M, McCoy K, Leung DH, Kloster M, Ramsey BR, Heltshe SH, Borowitz D. Pulmonary findings in infants with cystic fibrosis during the first year of life: Results from the Baby Observational and Nutrition Study (BONUS) cohort study. Pediatr Pulmonol. 2019 May;54(5):581-586. doi: 10.1002/ppul.24261. Epub 2019 Jan 22.

Reference Type: DERIVED

PMID: 30672141 (View on PubMed)

LeGrys VA, Moon TC, Laux J, Accurso F, Martiniano SA. A multicenter evaluation of sweat chloride concentration and variation in infants with cystic fibrosis. J Cyst Fibros. 2019 Mar;18(2):190-193. doi: 10.1016/j.jcf.2018.12.006. Epub 2018 Dec 21.

Reference Type: DERIVED

PMID: 30583934 (View on PubMed)

Kopp BT, Joseloff E, Goetz D, Ingram B, Heltshe SL, Leung DH, Ramsey BW, McCoy K, Borowitz D. Urinary metabolomics reveals unique metabolic signatures in infants with cystic fibrosis. J Cyst Fibros. 2019 Jul;18(4):507-515. doi: 10.1016/j.jcf.2018.10.016. Epub 2018 Nov 23.

Reference Type: DERIVED

PMID: 30477895 (View on PubMed)

Gelfond D, Heltshe SL, Skalland M, Heubi JE, Kloster M, Leung DH, Ramsey BW, Borowitz D; BONUS Study Investigators. Pancreatic Enzyme Replacement Therapy Use in Infants With Cystic Fibrosis Diagnosed by Newborn Screening. J Pediatr Gastroenterol Nutr. 2018 Apr;66(4):657-663. doi: 10.1097/MPG.0000000000001829.

Reference Type: DERIVED

PMID: 29176494 (View on PubMed)

Other Identifiers

R01DK095738

Identifier Type: NIH

Identifier Source: secondary_id

View Link

BONUS-IP-11

Identifier Type: -

Identifier Source: org_study_id