The EPIC Observational Study

NCT ID: NCT00676169

Last Updated: 2019-02-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

1248 participants

Study Classification

OBSERVATIONAL

Study Start Date

2004-10-31

Study Completion Date

2018-12-31

Brief Summary

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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

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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.

Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Observational

Pa negative or concurrently enrolled in the EPIC Clinical Trial

No interventions assigned to this group

Eligibility Criteria

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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.
Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cystic Fibrosis Foundation

OTHER

Sponsor Role collaborator

CF Therapeutics Development Network Coordinating Center

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Margaret Rosenfeld, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Ronald L. Gibson, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Wayne J. Morgan, MD

Role: PRINCIPAL_INVESTIGATOR

University of Arizona Health Sciences Center

Locations

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The University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Children's Hospital of Los Angeles / USC Medical School

Los Angeles, California, United States

Site Status

Kaiser Permanente Medical Center

Oakland, California, United States

Site Status

Packard Children's Hosp., Stanford University

Palo Alto, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

Children's Hospital Denver

Denver, Colorado, United States

Site Status

duPont Hospital for Children

Wilmington, Delaware, United States

Site Status

Nemours Children's Clinic

Jacksonville, Florida, United States

Site Status

All Children's Hospital CF Center

St. Petersburg, Florida, United States

Site Status

Emory University, Cystic Fibrosis Center

Atlanta, Georgia, United States

Site Status

Medical College of Georgia

Augusta, Georgia, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Riley Hospital, Indiana University

Indianapolis, Indiana, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Maine Medical Center

Portland, Maine, United States

Site Status

Children's Hospital, Boston

Boston, Massachusetts, United States

Site Status

University of Mass Memorial Health Care

Worcester, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Spectrum Health Hospitals - DeVos Children's Hospital

Grand Rapids, Michigan, United States

Site Status

Children's Hospitals & Clinics

Minneapolis, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Cardinal Glennon Children's Hospital - St. Louis University

St Louis, Missouri, United States

Site Status

Washington University School of Medicine/St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

University of Nebraska

Omaha, Nebraska, United States

Site Status

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Monmouth Medical Center

Long Branch, New Jersey, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Women & Children's Hospital of Buffalo

Buffalo, New York, United States

Site Status

Schneider Children's Hospital

New Hyde Park, New York, United States

Site Status

University of Rochester

Rochester, New York, United States

Site Status

SUNY Upstate Medical Center

Syracuse, New York, United States

Site Status

New York Medical College/Westchester Medical Center

Valhalla, New York, United States

Site Status

University of North Carolina, Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status

Rainbow Babies & Childrens Hospital

Cleveland, Ohio, United States

Site Status

Children's Hospital

Columbus, Ohio, United States

Site Status

Children's Medical Center

Dayton, Ohio, United States

Site Status

Oregon Health Sciences University

Portland, Oregon, United States

Site Status

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Rhode Island Hospital

Providence, Rhode Island, United States

Site Status

LeBonheur Children's Medical Center

Memphis, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Vermont Children's Hospital at Fletcher Allen Health Care

Burlington, Vermont, United States

Site Status

Children's Hospital & Regional Medical Center

Seattle, Washington, United States

Site Status

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

References

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Rosenfeld M, Emerson J, McNamara S, Thompson V, Ramsey BW, Morgan W, Gibson RL; EPIC Study Group. Risk factors for age at initial Pseudomonas acquisition in the cystic fibrosis epic observational cohort. J Cyst Fibros. 2012 Sep;11(5):446-53. doi: 10.1016/j.jcf.2012.04.003. Epub 2012 May 1.

Reference Type DERIVED
PMID: 22554417 (View on PubMed)

Rosenfeld M, Emerson J, McNamara S, Joubran K, Retsch-Bogart G, Graff GR, Gutierrez HH, Kanga JF, Lahiri T, Noyes B, Ramsey B, Ren CL, Schechter M, Morgan W, Gibson RL; EPIC Study Group Participating Clinical Sites. Baseline characteristics and factors associated with nutritional and pulmonary status at enrollment in the cystic fibrosis EPIC observational cohort. Pediatr Pulmonol. 2010 Sep;45(9):934-44. doi: 10.1002/ppul.21279.

Reference Type DERIVED
PMID: 20597081 (View on PubMed)

Treggiari MM, Rosenfeld M, Mayer-Hamblett N, Retsch-Bogart G, Gibson RL, Williams J, Emerson J, Kronmal RA, Ramsey BW; EPIC Study Group. Early anti-pseudomonal acquisition in young patients with cystic fibrosis: rationale and design of the EPIC clinical trial and observational study'. Contemp Clin Trials. 2009 May;30(3):256-68. doi: 10.1016/j.cct.2009.01.003. Epub 2009 Jan 15.

Reference Type DERIVED
PMID: 19470318 (View on PubMed)

Other Identifiers

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EPIC002

Identifier Type: -

Identifier Source: org_study_id

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