The EPIC Observational Study

Cystic Fibrosis Clinical Trial

— EPIC OBS  

Official title:

Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas Aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children With CF

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00676169
• Other study ID #: EPIC002
• Status: Completed
• Start date: October 2004
• Est. completion date: December 2018

Study information

• Verified date: January 2019
• Source: CF Therapeutics Development Network Coordinating Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 1248
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis

Locations

Country	Name	City	State
United States	Children's Hospital Medical Center of Akron	Akron	Ohio
United States	Albany Medical College	Albany	New York
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory University, Cystic Fibrosis Center	Atlanta	Georgia
United States	Medical College of Georgia	Augusta	Georgia
United States	The University of Alabama at Birmingham	Birmingham	Alabama
United States	Children's Hospital, Boston	Boston	Massachusetts
United States	Women & Children's Hospital of Buffalo	Buffalo	New York
United States	Vermont Children's Hospital at Fletcher Allen Health Care	Burlington	Vermont
United States	University of North Carolina, Chapel Hill	Chapel Hill	North Carolina
United States	Children's Memorial Hospital	Chicago	Illinois
United States	Rainbow Babies & Childrens Hospital	Cleveland	Ohio
United States	Children's Hospital	Columbus	Ohio
United States	Children's Medical Center	Dayton	Ohio
United States	Children's Hospital Denver	Denver	Colorado
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Spectrum Health Hospitals - DeVos Children's Hospital	Grand Rapids	Michigan
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	Texas Children's Hospital	Houston	Texas
United States	Riley Hospital, Indiana University	Indianapolis	Indiana
United States	University of Iowa	Iowa City	Iowa
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	Nemours Children's Clinic	Jacksonville	Florida
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Dartmouth-Hitchcock Medical Center	Lebanon	New Hampshire
United States	University of Kentucky	Lexington	Kentucky
United States	Monmouth Medical Center	Long Branch	New Jersey
United States	Children's Hospital of Los Angeles / USC Medical School	Los Angeles	California
United States	University of Wisconsin Hospital and Clinics	Madison	Wisconsin
United States	LeBonheur Children's Medical Center	Memphis	Tennessee
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Children's Hospitals & Clinics	Minneapolis	Minnesota
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Schneider Children's Hospital	New Hyde Park	New York
United States	Kaiser Permanente Medical Center	Oakland	California
United States	University of Nebraska	Omaha	Nebraska
United States	Packard Children's Hosp., Stanford University	Palo Alto	California
United States	St. Christopher's Hospital for Children	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Maine Medical Center	Portland	Maine
United States	Oregon Health Sciences University	Portland	Oregon
United States	Rhode Island Hospital	Providence	Rhode Island
United States	University of Rochester	Rochester	New York
United States	Cardinal Glennon Children's Hospital - St. Louis University	Saint Louis	Missouri
United States	Washington University School of Medicine/St. Louis Children's Hospital	Saint Louis	Missouri
United States	All Children's Hospital CF Center	Saint Petersburg	Florida
United States	University of Utah	Salt Lake City	Utah
United States	University of California, San Francisco	San Francisco	California
United States	Children's Hospital & Regional Medical Center	Seattle	Washington
United States	SUNY Upstate Medical Center	Syracuse	New York
United States	New York Medical College/Westchester Medical Center	Valhalla	New York
United States	duPont Hospital for Children	Wilmington	Delaware
United States	University of Mass Memorial Health Care	Worcester	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
CF Therapeutics Development Network Coordinating Center	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Secondary	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
Secondary	To describe temporal changes in anti-pseudomonal serology and airway microbiology.		over the two-to-five year observational period
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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