Clinical trial NCT01973192: Viral Pathogenesis of Early Cystic Fibrosis Lung Disease
Official title: Viral Pathogenesis of Early Cystic Fibrosis Lung Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Lead sponsor: Indiana University
Sponsor class: OTHER
Collaborators: name: National Institutes of Health (NIH)
name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2013-10-31
Enrollment: 65
Sex: ALL
Minimum age (years): 2
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants, less than 4 months of age who have been diagnosed with Cystic Fibrosis

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Viral infection
timeFrame: 12 months
Secondary outcomes:
measure: Pulmonary exacerbation rate
timeFrame: 12 Months
measure: Forced Expiratory Volume
timeFrame: 12 months
Other outcomes:
measure: Bronchiectasis
timeFrame: 12 Months
Locations:
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University Health
state: Indiana
city: St. Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Melbourne
country: Australia
facility: The Royal Children's Hospital
state: Victoria
city: West Perth
country: Australia
facility: Telethon Kids Institute
Study officials:
affiliation: Indiana University School of Medicine
name: Stephanie D. Davis, MD
role: PRINCIPAL_INVESTIGATOR
References: