NCT00676169

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,248

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Oct 2004

Longer than P75 for all trials

Geographic Reach
1 country

54 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2004

Completed
3.6 years until next milestone

First Submitted

Initial submission to the registry

May 8, 2008

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 12, 2008

Completed
10.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2018

Completed
Last Updated

February 1, 2019

Status Verified

January 1, 2019

Enrollment Period

14.2 years

First QC Date

May 8, 2008

Last Update Submit

January 30, 2019

Conditions

Keywords

Pseudomonas aeruginosaEPIC

Outcome Measures

Primary Outcomes (2)

  • 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

  • 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 Outcomes (7)

  • 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

  • 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 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

  • +2 more secondary outcomes

Study Arms (1)

Observational

Pa negative or concurrently enrolled in the EPIC Clinical Trial

Eligibility Criteria

AgeUp to 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

Children with Cystic Fibrosis who are Pa negative or concurrently enrolled in the EPIC Clinical Trial

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (54)

The University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Children's Hospital of Los Angeles / USC Medical School

Los Angeles, California, 90027, United States

Location

Kaiser Permanente Medical Center

Oakland, California, 94611, United States

Location

Packard Children's Hosp., Stanford University

Palo Alto, California, 94304, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Children's Hospital Denver

Denver, Colorado, 80218, United States

Location

duPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

Location

All Children's Hospital CF Center

St. Petersburg, Florida, 33701, United States

Location

Emory University, Cystic Fibrosis Center

Atlanta, Georgia, 30322, United States

Location

Medical College of Georgia

Augusta, Georgia, 30912, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

Riley Hospital, Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kentucky

Lexington, Kentucky, 40536, United States

Location

Maine Medical Center

Portland, Maine, 04102, United States

Location

Children's Hospital, Boston

Boston, Massachusetts, 02115, United States

Location

University of Mass Memorial Health Care

Worcester, Massachusetts, 01655, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Spectrum Health Hospitals - DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Children's Hospitals & Clinics

Minneapolis, Minnesota, 55404, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Cardinal Glennon Children's Hospital - St. Louis University

St Louis, Missouri, 63104, United States

Location

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

St Louis, Missouri, 63310, United States

Location

University of Nebraska

Omaha, Nebraska, 68178, United States

Location

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Monmouth Medical Center

Long Branch, New Jersey, 07740, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

Women & Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

Schneider Children's Hospital

New Hyde Park, New York, 11040, United States

Location

University of Rochester

Rochester, New York, 14642, United States

Location

SUNY Upstate Medical Center

Syracuse, New York, 13210, United States

Location

New York Medical College/Westchester Medical Center

Valhalla, New York, 10595, United States

Location

University of North Carolina, Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States

Location

Rainbow Babies & Childrens Hospital

Cleveland, Ohio, 44106, United States

Location

Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Medical Center

Dayton, Ohio, 45404, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02905, United States

Location

LeBonheur Children's Medical Center

Memphis, Tennessee, 38103, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Vermont Children's Hospital at Fletcher Allen Health Care

Burlington, Vermont, 05401, United States

Location

Children's Hospital & Regional Medical Center

Seattle, Washington, 98105, United States

Location

University of Wisconsin Hospital and Clinics

Madison, Wisconsin, 53792, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (3)

  • 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.

  • 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.

  • 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.

Biospecimen

Retention: SAMPLES WITH DNA

For study purposes, OP swabs or expectorated sputum for bacterial culture will be obtained annually beginning in the calendar year of first isolation of Pa from a respiratory culture at the local site laboratory. A serum sample for serology assessment and banking will be obtained annually in conjunction with a scheduled clinical blood draw whenever possible. Under a separate consent, additional blood (6 ml) will be collected for DNA use and banking for studies of genetic factors that may be associated with CF pathogenesis, disease progression, and clinical outcomes. These studies will test for association between gene variants and various CF-related phenotypes using either a targeted (i.e., candidate gene) approach or by performing a whole-genome scan.

MeSH Terms

Conditions

Cystic FibrosisPseudomonas Infections

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesGram-Negative Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Study Officials

  • 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

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 8, 2008

First Posted

May 12, 2008

Study Start

October 1, 2004

Primary Completion

December 1, 2018

Study Completion

December 1, 2018

Last Updated

February 1, 2019

Record last verified: 2019-01

Locations