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Viral Pathogenesis of Early Cystic Fibrosis Lung Disease (Early CF)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01973192
Recruitment Status : Completed
First Posted : October 31, 2013
Last Update Posted : April 4, 2017
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Stephanie D. Davis, Indiana University

Brief Summary:
The purpose of this study is to test the hypothesis that early viral infections alter the bacterial flora and inflammatory profile in the airway and accelerate progression of pulmonary disease in infants with cystic fibrosis.

Condition or disease
Cystic Fibrosis

Detailed Description:
The proposed study is a unique international collaboration between three large CF research centers. This proposal will determine the impact of early respiratory viral infections on bacterial flora and inflammatory profiles in the CF airway as well as the impact of these pathogens on clinical, physiologic and structural markers of disease.The proposed study is designed to follow infants diagnosed with CF through newborn screening to determine the effect of viral infections on the lower airway microbiome, clinical symptoms, pulmonary function and structural changes during the first year of life. The proposed study will measure lower airway inflammation and infection using BAL, oral swabs, and nasal swabs; outcomes will be assessed through infant lung function testing, computerized tomography scans of the chest, and pulmonary exacerbation rate.

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Study Type : Observational
Actual Enrollment : 65 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Viral Pathogenesis of Early Cystic Fibrosis Lung Disease
Study Start Date : May 2013
Actual Primary Completion Date : December 1, 2016
Actual Study Completion Date : December 1, 2016

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Viral infection [ Time Frame: 12 months ]
    To determine the effect(s) of viral infections on the evolution of endobronchial bacterial infection and inflammation in CF infants.

Secondary Outcome Measures :
  1. Pulmonary exacerbation rate [ Time Frame: 12 Months ]
    To identify the impact of respiratory viruses on the onset, frequency, and duration of respiratory symptoms in CF infants diagnosed through newborn screening.

  2. Forced Expiratory Volume [ Time Frame: 12 months ]
    To assess development of early lung disease as defined through physiological measures of forced expiratory flows, lung volumes, and ventilation inhomogeneity in CF infants.

Other Outcome Measures:
  1. Bronchiectasis [ Time Frame: 12 Months ]
    To evaluate the association of early viruses on the development of early lung disease in CF infants as defined through comprehensive structural and airway modeling techniques.

Biospecimen Retention:   Samples With DNA
Blood Urea, Bronchopulmonary Lavage samples, Nasal swabs and Oral swabs, Stool samples

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Months to 4 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Infants, less than 4 months of age who have been diagnosed with Cystic Fibrosis

Inclusion Criteria:

  1. Diagnosis of CF by newborn screening, at least one clinical feature of CF, and documented sweat chloride greater than 60 mEq/L by quantitative pilocarpine iontophoresis or compatible genotype with two identifiable mutant CFTR alleles.
  2. Less than 4 months of age at Screening Visit
  3. Ability to comply with study visits and study procedures as judged by site investigator.

Exclusion Criteria:

  1. Intercurrent respiratory illness, defined as increase in cough, wheezing, or respiratory rate with onset 14 days before iPFT-bronchoscopy visit.
  2. Measured hemoglobin oxygen saturation less than 95% during the iPFT-bronchoscopy visit.
  3. History of adverse reaction to sedation.
  4. Clinically significant upper airway obstruction as determined by the site investigator.
  5. Severe gastroesophageal reflux, defined as persistent frequent emesis despite therapy.
  6. Major organ dysfunction, not including pancreatic dysfunction.
  7. Physical findings that would compromise the safety of the subject or the quality of the study data as determined by site investigator.

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 identifier (NCT number): NCT01973192

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United States, Indiana
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States, 46202
United States, Missouri
St. Louis Children's Hospital
St. Louis, Missouri, United States, 63110
Australia, Victoria
The Royal Children's Hospital
Melbourne, Victoria, Australia
Telethon Kids Institute
West Perth, Australia, 6872
Sponsors and Collaborators
Indiana University School of Medicine
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
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Principal Investigator: Stephanie D. Davis, MD Indiana University School of Medicine
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Responsible Party: Stephanie D. Davis, MD, Section Director of Pediatric Pulmonology, Allergy and Sleep Medicine, Indiana University Identifier: NCT01973192    
Other Study ID Numbers: 1R01HL116211-01 ( U.S. NIH Grant/Contract )
1R01HL116211-01 ( U.S. NIH Grant/Contract )
First Posted: October 31, 2013    Key Record Dates
Last Update Posted: April 4, 2017
Last Verified: April 2017

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Lung Diseases
Pulmonary Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases