The EPIC Observational Study
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT00676169
- Lead Sponsor
- CF Therapeutics Development Network Coordinating Center
- Brief Summary
The purpose of this study is to better define risk factors preceding first isolation of Pseudomonas aeruginosa (Pa) from respiratory cultures in cystic fibrosis (CF) lung disease and to better define clinical outcomes associated with acquisition of Pa. This study will also collect and bank DNA samples for current and future studies designed to enhance the understanding of the pathogenesis of CF.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1248
- 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.
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To better define clinical outcomes associated with acquisition of Pa, as well as outcomes associated with emergence of mucoid Pa and antibiotic-resistant Pa. over the two-to-five-year observational period To better define risk factors for first isolation of Pa from respiratory culture, as well as for emergence of mucoid Pa and antibiotic-resistant Pa. over the two-to-five-year observational period
- Secondary Outcome Measures
Name Time Method To better define clinical outcomes associated with isolation of S. aureus from respiratory cultures, as well as outcomes associated with emergence of methicillin-resistant S. aureus (MRSA). over the two-to-five year observational period To provide a cohort of subjects who acquire Pa during the observational study period but who do not enroll in EPIC Clinical Trial and therefore receive non protocol-based anti-pseudomonal therapy. over the two-to-five year observational period To bank Pa and S. aureus isolates and serum samples for future studies to enhance the understanding of early CF lung disease. over the two-to-five year observational period To use and bank DNA samples for analyses of genetic factors that may be associated with CF pathogenesis, disease progression, and clinical outcomes. over the two-to-five year observational period For subjects who enroll in EPIC Clinical Trial, to collect ancillary data on risk factors preceding trial enrollment and to provide follow-up for clinical endpoints after trial participation has ended. over the two-to-five year observational period Among subjects who acquire Pa but do not enroll in the EPIC Clinical Trial, to examine the effect of the duration of Pa positive respiratory cultures prior to initiation of anti-pseudomonal therapy and the type and length of anti-pseudomonal therapy. over the two-to-five year observational period To describe temporal changes in anti-pseudomonal serology and airway microbiology. over the two-to-five year observational period
Related Research Topics
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Trial Locations
- Locations (54)
Emory University, Cystic Fibrosis Center
🇺🇸Atlanta, Georgia, United States
Children's Hospital
🇺🇸Columbus, Ohio, United States
Rainbow Babies & Childrens Hospital
🇺🇸Cleveland, Ohio, United States
Children's Hospital, Boston
🇺🇸Boston, Massachusetts, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Children's Hospital & Regional Medical Center
🇺🇸Seattle, Washington, United States
Kaiser Permanente Medical Center
🇺🇸Oakland, California, United States
Riley Hospital, Indiana University
🇺🇸Indianapolis, Indiana, United States
duPont Hospital for Children
🇺🇸Wilmington, Delaware, United States
Children's Hospital of Los Angeles / USC Medical School
🇺🇸Los Angeles, California, United States
Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
Packard Children's Hosp., Stanford University
🇺🇸Palo Alto, California, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
All Children's Hospital CF Center
🇺🇸Saint Petersburg, Florida, United States
Medical College of Georgia
🇺🇸Augusta, Georgia, United States
Nemours Children's Clinic
🇺🇸Jacksonville, Florida, United States
Children's Hospital Medical Center of Akron
🇺🇸Akron, Ohio, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
Monmouth Medical Center
🇺🇸Long Branch, New Jersey, United States
Dartmouth-Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Maine Medical Center
🇺🇸Portland, Maine, United States
University of Mass Memorial Health Care
🇺🇸Worcester, Massachusetts, United States
Spectrum Health Hospitals - DeVos Children's Hospital
🇺🇸Grand Rapids, Michigan, United States
Children's Hospitals & Clinics
🇺🇸Minneapolis, Minnesota, United States
Vermont Children's Hospital at Fletcher Allen Health Care
🇺🇸Burlington, Vermont, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Washington University School of Medicine/St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
Albany Medical College
🇺🇸Albany, New York, United States
Women & Children's Hospital of Buffalo
🇺🇸Buffalo, New York, United States
University of North Carolina, Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Schneider Children's Hospital
🇺🇸New Hyde Park, New York, United States
University of Rochester
🇺🇸Rochester, New York, United States
New York Medical College/Westchester Medical Center
🇺🇸Valhalla, New York, United States
SUNY Upstate Medical Center
🇺🇸Syracuse, New York, United States
LeBonheur Children's Medical Center
🇺🇸Memphis, Tennessee, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
St. Christopher's Hospital for Children
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Children's Medical Center
🇺🇸Dayton, Ohio, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
The University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
University of Nebraska
🇺🇸Omaha, Nebraska, United States
Oregon Health Sciences University
🇺🇸Portland, Oregon, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Children's Hospital Denver
🇺🇸Denver, Colorado, United States
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States