EPIC OBS: The EPIC Observational Study

• Sponsor: CF Therapeutics Development Network Coordinating Center (Other)
• Overall Status: Completed
• CT.gov ID: NCT00676169
• Collaborator: Cystic Fibrosis Foundation (Other)
• Enrollment: 1,248
• Locations: 54
• Duration (Months): 170
• Patients Per Site: 23.1
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis

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.

Study Design

• Study Type: Observational
• Actual Enrollment: 1248 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas Aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children With CF
• Study Start Date: Oct 1, 2004
• Actual Primary Completion Date: Dec 1, 2018
• Actual Study Completion Date: Dec 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Observational Pa negative or concurrently enrolled in the EPIC Clinical Trial

Outcome Measures

Primary Outcome Measures

1. 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]

2. 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 Outcome Measures

1. 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]

2. To describe temporal changes in anti-pseudomonal serology and airway microbiology. [over the two-to-five year observational period]

3. 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]

4. 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]

5. 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]

6. 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]

7. 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]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 12 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The University of Alabama at Birmingham	Birmingham	Alabama	United States	35233
2	Children's Hospital of Los Angeles / USC Medical School	Los Angeles	California	United States	90027
3	Kaiser Permanente Medical Center	Oakland	California	United States	94611
4	Packard Children's Hosp., Stanford University	Palo Alto	California	United States	94304
5	University of California, San Francisco	San Francisco	California	United States	94143
6	Children's Hospital Denver	Denver	Colorado	United States	80218
7	duPont Hospital for Children	Wilmington	Delaware	United States	19803
8	Nemours Children's Clinic	Jacksonville	Florida	United States	32207
9	All Children's Hospital CF Center	Saint Petersburg	Florida	United States	33701
10	Emory University, Cystic Fibrosis Center	Atlanta	Georgia	United States	30322
11	Medical College of Georgia	Augusta	Georgia	United States	30912
12	Children's Memorial Hospital	Chicago	Illinois	United States	60614
13	Riley Hospital, Indiana University	Indianapolis	Indiana	United States	46202
14	University of Iowa	Iowa City	Iowa	United States	52242
15	University of Kentucky	Lexington	Kentucky	United States	40536
16	Maine Medical Center	Portland	Maine	United States	04102
17	Children's Hospital, Boston	Boston	Massachusetts	United States	02115
18	University of Mass Memorial Health Care	Worcester	Massachusetts	United States	01655
19	University of Michigan	Ann Arbor	Michigan	United States	48109
20	Children's Hospital of Michigan	Detroit	Michigan	United States	48201
21	Spectrum Health Hospitals - DeVos Children's Hospital	Grand Rapids	Michigan	United States	49503
22	Children's Hospitals & Clinics	Minneapolis	Minnesota	United States	55404
23	University of Mississippi Medical Center	Jackson	Mississippi	United States	39216
24	Children's Mercy Hospital	Kansas City	Missouri	United States	64108
25	Cardinal Glennon Children's Hospital - St. Louis University	Saint Louis	Missouri	United States	63104
26	Washington University School of Medicine/St. Louis Children's Hospital	Saint Louis	Missouri	United States	63310
27	University of Nebraska	Omaha	Nebraska	United States	68178
28	Dartmouth-Hitchcock Medical Center	Lebanon	New Hampshire	United States	03756
29	Monmouth Medical Center	Long Branch	New Jersey	United States	07740
30	Albany Medical College	Albany	New York	United States	12208
31	Women & Children's Hospital of Buffalo	Buffalo	New York	United States	14222
32	Schneider Children's Hospital	New Hyde Park	New York	United States	11040
33	University of Rochester	Rochester	New York	United States	14642
34	SUNY Upstate Medical Center	Syracuse	New York	United States	13210
35	New York Medical College/Westchester Medical Center	Valhalla	New York	United States	10595
36	University of North Carolina, Chapel Hill	Chapel Hill	North Carolina	United States	27599
37	Children's Hospital Medical Center of Akron	Akron	Ohio	United States	44308
38	Rainbow Babies & Childrens Hospital	Cleveland	Ohio	United States	44106
39	Children's Hospital	Columbus	Ohio	United States	43205
40	Children's Medical Center	Dayton	Ohio	United States	45404
41	Oregon Health Sciences University	Portland	Oregon	United States	97239
42	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania	United States	17033
43	St. Christopher's Hospital for Children	Philadelphia	Pennsylvania	United States	19134
44	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania	United States	15213
45	Rhode Island Hospital	Providence	Rhode Island	United States	02905
46	LeBonheur Children's Medical Center	Memphis	Tennessee	United States	38103
47	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
48	Cook Children's Medical Center	Fort Worth	Texas	United States	76104
49	Texas Children's Hospital	Houston	Texas	United States	77030
50	University of Utah	Salt Lake City	Utah	United States	84132
51	Vermont Children's Hospital at Fletcher Allen Health Care	Burlington	Vermont	United States	05401
52	Children's Hospital & Regional Medical Center	Seattle	Washington	United States	98105
53	University of Wisconsin Hospital and Clinics	Madison	Wisconsin	United States	53792
54	Children's Hospital of Wisconsin	Milwaukee	Wisconsin	United States	53226

Sponsors and Collaborators

• CF Therapeutics Development Network Coordinating Center
• Cystic Fibrosis Foundation

Investigators

• Principal Investigator: Margaret Rosenfeld, MD, MPH, Seattle Children's Hospital
• Principal Investigator: Ronald L. Gibson, MD, PhD, Seattle Children's Hospital
• Principal Investigator: Wayne J. Morgan, MD, University of Arizona Health Sciences Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: CF Therapeutics Development Network Coordinating Center
• ClinicalTrials.gov Identifier: NCT00676169
• Other Study ID Numbers: EPIC002
• First Posted: May 12, 2008
• Last Update Posted: Feb 1, 2019
• Last Verified: Jan 1, 2019

• Keywords provided by CF Therapeutics Development Network Coordinating Center: Pseudomonas aeruginosa; EPIC
• Additional relevant MeSH terms: Cystic Fibrosis

Study Results

No Results Posted as of Feb 1, 2019