NCT01424696

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
231

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Dec 2011

Typical duration for all trials

Geographic Reach
1 country

28 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 25, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 29, 2011

Completed
3 months until next milestone

Study Start

First participant enrolled

December 1, 2011

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2015

Completed
Last Updated

September 28, 2015

Status Verified

September 1, 2015

Enrollment Period

3.3 years

First QC Date

August 25, 2011

Last Update Submit

September 25, 2015

Conditions

Keywords

growth measuresnutritional statuspulmonary infectioninflammatory markerspancreatic enzyme replacement therapy

Outcome Measures

Primary Outcomes (1)

  • 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

Study Arms (1)

Cystic Fibrosis in 1st 3 months of life

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

Eligibility Criteria

AgeUp to 3 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

Infants diagnosed with CF who attend regular clinic visits during the first year of life

You may qualify if:

  • Signed informed consent
  • Males or females no more than three and one half (3.5) months of age at enrollment
  • Documentation of a CF diagnosis as evidenced by:
  • 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
  • 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
  • Enrolled in the Cystic Fibrosis Foundation Patient Registry. (Patients may enroll in the Registry at Enrollment Visit if not previously enrolled.)

You may not qualify if:

  • Children unable to take full oral feeds
  • 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.
  • Gestational age less than 35 weeks and/or birth weight \< 2.5 kg.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Emory CF Center

Atlanta, Georgia, 30322, United States

Location

Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Iowa City University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Helen DeVos Children's Hospital CF Care Center

Grand Rapids, Michigan, 49503, United States

Location

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

State University of New York at Buffalo

Buffalo, New York, 14222, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Vanderbilt CF Center

Nashville, Tennessee, 37232, United States

Location

Austin Children's Chest Associates

Austin, Texas, 78723, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Related Publications (5)

  • 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.

  • 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.

  • 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.

  • 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.

  • 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.

Biospecimen

Retention: SAMPLES WITH DNA

* serum and/or plasma * urine * stool * oropharyngeal swab (OP swab) * saliva * sweat * buccal brushings

MeSH Terms

Conditions

Cystic FibrosisFailure to ThriveExocrine Pancreatic Insufficiency

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

August 25, 2011

First Posted

August 29, 2011

Study Start

December 1, 2011

Primary Completion

April 1, 2015

Study Completion

April 1, 2015

Last Updated

September 28, 2015

Record last verified: 2015-09

Locations