Cystic FibrosisBONUSNCT01424696
Baby Observational and Nutritional Study
An observational study of Cystic Fibrosis and Growth Failure, run by Seattle Children's Hospital at 28 sites in United States.
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. Read more about this study
- Who is it for?
- Ages Up to 3 months
- Conditions the study is for
- Cystic Fibrosis, Growth Failure, Exocrine Pancreatic Insufficiency
- How many people
- 231 participants
- Where
- 28 sitesUnited States
- Study dates
- Dec 2011to Apr 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 3 months
- All sexes
- No healthy volunteers
- Child
Study population: Infants diagnosed with CF who attend regular clinic visits during the first year of life
Who may be able to join
- 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.)
Who cannot join
- 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.
Trial locations and contacts
28 sites in 1 country.
28 sites in 1 country
United States28 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory CF Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Austin Children's Chest Associates
Austin, Texas, 78723
University of Alabama at Birmingham
Birmingham, Alabama, 35233
State University of New York at Buffalo
Buffalo, New York, 14222
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390
Cook Children's Medical Center
Fort Worth, Texas, 76104
Helen DeVos Children's Hospital CF Care Center
Grand Rapids, Michigan, 49503
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Iowa City University of Iowa
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404
Vanderbilt CF Center
Nashville, Tennessee, 37232
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84108
Seattle Children's Hospital
Seattle, Washington, 98105
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104
Washington University School of Medicine
St Louis, Missouri, 63110
SUNY Upstate Medical University
Syracuse, New York, 13210
About this study
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.
Read the 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.
- Keywords
- growth measures, nutritional status, pulmonary infection, inflammatory markers, pancreatic enzyme replacement therapy
- Medical subject headings
- Cystic Fibrosis, Failure to Thrive, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystic Fibrosis in 1st 3 months of life | Early Diagnosis: Children diagnosed with CF in first 3 months of life |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2011 (estimated)
- Study startDec 2011
- Primary completionApr 2015
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 231 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seattle Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Responsible party
- Principal investigator, Bonnie Ramsey, Seattle Children's Hospital
- Investigators
- Drucy Borowitz, MD, Principal investigator, State University of New York at BuffaloDaniel Leung, MD, Principal investigator, Baylor College of MedicineJames Heubi, MD, Principal investigator, University of CincinnatiDaniel Gelfond, MD, Principal investigator, Women & Children's Hospital of Buffalo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01424696
- Sponsor's protocol ID
- BONUS-IP-11
- Other IDs
- R01DK095738
- Acronym
- BONUS
- Status last verified
- Sep 2015
- First submitted
- 25 Aug 2011
- Last updated
- 28 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 34016873Goetz 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.
PubMed 30672141LeGrys 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.
PubMed 30583934Kopp 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.
PubMed 30477895Gelfond 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.
PubMed 29176494
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Baby Observational and Nutritional Study (BONUS)
- ClinicalTrials.gov ID
- NCT01424696
- Acronym / sponsor ID
- BONUS / BONUS-IP-11
- Keywords
- growth measures, nutritional status, pulmonary infection, inflammatory markers, pancreatic enzyme replacement therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 231 participants (actual)
- Lead sponsor
- Seattle Children's HospitalUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Dec 2011
- Primary completion
- Apr 2015 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 29 Aug 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cystic Fibrosis in 1st 3 months of life | – | –Early Diagnosis: Children diagnosed with CF in first 3 months of life |
Who may qualify
- Ages
- Up to 3 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants diagnosed with CF who attend regular clinic visits during the first year of life
Key inclusion criteria (7 listed)
- 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:
- AND
- Enrolled in the Cystic Fibrosis Foundation Patient Registry. (Patients may enroll in the Registry at Enrollment Visit if not previously enrolled.)
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2015.
- Derived from this study: PubMed 34016873Sathe 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.
- Derived from this study: PubMed 30672141Goetz 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.
- Derived from this study: PubMed 30583934LeGrys 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.
- Derived from this study: PubMed 30477895Kopp 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.
- Derived from this study: PubMed 29176494Gelfond 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Drucy Borowitz, MD principal investigator
State University of New York at Buffalo
Daniel Leung, MD principal investigator
Baylor College of Medicine
James Heubi, MD principal investigator
University of Cincinnati
28 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis and Growth Failure, run by Seattle Children's Hospital at 28 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incremental gain in weight, length, and head circumference (time frame: one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01424696, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2015.