The EPIC Observational Study

The EPIC Observational Study is a longitudinal, prospective, observational study that was
originally conducted at 59 sites. The current five-year extension study is being conducted at
54 sites.

The EPIC Observational Study will serve as a freestanding epidemiologic study of the risk
factors for and clinical impact of initial Pa acquisition and anti-pseudomonal therapy.
Defining the risk factors for Pa acquisition can potentially allow for preventive measures
and identification of high-risk populations requiring closer monitoring. Despite rigorous
data collection, previous studies have been limited by small sample sizes and by conduct at
one or two centers. This study will include a much larger sample size from many more centers
than previous studies. It will thus provide for more generalizable results and more precise
risk estimates for previously identified risk factors for Pa acquisition, and it will allow
for exploration of novel risk factors not included in earlier studies. Better understanding
of the clinical outcomes associated with Pa acquisition and the outcomes associated with
different types of anti-pseudomonal therapies will inform the development of rational early
intervention treatment regimens. Better knowledge about temporal relationships between
respiratory signs and symptoms, Pa serology, and CF airway microbiology may lead to improved
strategies for early detection of Pa and could have important implications for the timing of
interventions aimed at preventing or treating early Pa acquisition. Finally, this study will
serve as an important source of Pa and S. aureus isolates, serum samples, and DNA samples
that will be used and banked for studies designed to enhance the understanding of the
pathogenesis of CF, e.g., microarray investigations of early Pa isolates, investigations to
identify proteomic biomarkers of airway inflammation, and investigations to identify genetic
factors related to CF disease progression, including early lung disease, and clinical
outcomes.

Inclusion Criteria:

- Male or female ages less than or equal to 12 years.

- Diagnosis of CF based upon the criteria established by the 1997 CF Consensus
Conference: (i) sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis;
or (ii) genotype with two identifiable mutations consistent with CF; or (iii) an
abnormal nasal transepithelial potential difference, and (iv) one or more clinical
features consistent with CF.

- No prior isolation of Pa from respiratory cultures (1 or more cultures in 24 months
prior to enrollment), or, if prior isolation of Pa from respiratory cultures, at least
a two-year history of Pa negative cultures (1 or more cultures/year), or concurrently
enrolled in the EPIC Clinical Trial.

- Signed informed consent to participate in data submission to the CFF National Patient
Registry.

- Signed informed consent by parent or legal guardian.