Cystic FibrosisEPIC OBSNCT00676169
The EPIC Observational Study
An observational study of Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 54 sites in United States.
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. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 1,248 participants
- Where
- 54 sitesUnited States
- Study dates
- Oct 2004to Dec 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with Cystic Fibrosis who are Pa negative or concurrently enrolled in the EPIC Clinical Trial
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
54 sites in 1 country.
54 sites in 1 country
United States54 sites
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Albany Medical College
Albany, New York, 12208
University of Michigan
Ann Arbor, Michigan, 48109
Emory University, Cystic Fibrosis Center
Atlanta, Georgia, 30322
Medical College of Georgia
Augusta, Georgia, 30912
The University of Alabama at Birmingham
Birmingham, Alabama, 35233
Children's Hospital, Boston
Boston, Massachusetts, 02115
Women & Children's Hospital of Buffalo
Buffalo, New York, 14222
Vermont Children's Hospital at Fletcher Allen Health Care
Burlington, Vermont, 05401
University of North Carolina, Chapel Hill
Chapel Hill, North Carolina, 27599
Children's Memorial Hospital
Chicago, Illinois, 60614
Rainbow Babies & Childrens Hospital
Cleveland, Ohio, 44106
Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center
Dayton, Ohio, 45404
Children's Hospital Denver
Denver, Colorado, 80218
Children's Hospital of Michigan
Detroit, Michigan, 48201
Cook Children's Medical Center
Fort Worth, Texas, 76104
Spectrum Health Hospitals - DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital, Indiana University
Indianapolis, Indiana, 46202
University of Iowa
Iowa City, Iowa, 52242
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Nemours Children's Clinic
Jacksonville, Florida, 32207
Children's Mercy Hospital
Kansas City, Missouri, 64108
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Kentucky
Lexington, Kentucky, 40536
Monmouth Medical Center
Long Branch, New Jersey, 07740
Children's Hospital of Los Angeles / USC Medical School
Los Angeles, California, 90027
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792
LeBonheur Children's Medical Center
Memphis, Tennessee, 38103
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospitals & Clinics
Minneapolis, Minnesota, 55404
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Schneider Children's Hospital
New Hyde Park, New York, 11040
Kaiser Permanente Medical Center
Oakland, California, 94611
University of Nebraska
Omaha, Nebraska, 68178
Packard Children's Hosp., Stanford University
Palo Alto, California, 94304
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Maine Medical Center
Portland, Maine, 04102
Oregon Health Sciences University
Portland, Oregon, 97239
Rhode Island Hospital
Providence, Rhode Island, 02905
University of Rochester
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
University of California, San Francisco
San Francisco, California, 94143
Children's Hospital & Regional Medical Center
Seattle, Washington, 98105
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, 63104
Washington University School of Medicine/St. Louis Children's Hospital
St Louis, Missouri, 63310
All Children's Hospital CF Center
St. Petersburg, Florida, 33701
SUNY Upstate Medical Center
Syracuse, New York, 13210
New York Medical College/Westchester Medical Center
Valhalla, New York, 10595
duPont Hospital for Children
Wilmington, Delaware, 19803
University of Mass Memorial Health Care
Worcester, Massachusetts, 01655
About this study
Official title: Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas Aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children With CF
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Keywords
- Pseudomonas aeruginosa, EPIC
- Medical subject headings
- Cystic Fibrosis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Observational | Pa negative or concurrently enrolled in the EPIC Clinical Trial |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: over the two-to-five year observational period
To describe temporal changes in anti-pseudomonal serology and airway microbiology.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: over the two-to-five year observational period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 May 2008 (estimated)
- Study startOct 2004
- Primary completionDec 2018
- Study completionDec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,248 participants (actual)
Sponsor and investigators
- Lead sponsor
- CF Therapeutics Development Network Coordinating Center
- Sponsor type
- Research network
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Margaret Rosenfeld, MD, MPH, Principal investigator, Seattle Children's HospitalRonald L. Gibson, MD, PhD, Principal investigator, Seattle Children's HospitalWayne J. Morgan, MD, Principal investigator, University of Arizona Health Sciences Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00676169
- Sponsor's protocol ID
- EPIC002
- Acronym
- EPIC OBS
- Status last verified
- Jan 2019
- First submitted
- 8 May 2008
- Last updated
- 1 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 22554417Rosenfeld 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.
PubMed 20597081Treggiari 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.
PubMed 19470318
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas Aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children With CF
- ClinicalTrials.gov ID
- NCT00676169
- Acronym / sponsor ID
- EPIC OBS / EPIC002
- Conditions
- Cystic Fibrosis
- Keywords
- Pseudomonas aeruginosa, EPIC
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,248 participants (actual)
- Lead sponsor
- CF Therapeutics Development Network Coordinating CenterResearch network
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Oct 2004
- Primary completion
- Dec 2018 (actual)
- Study completion
- Dec 2018 (actual)
- First posted
- 12 May 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational | – | –Pa negative or concurrently enrolled in the EPIC Clinical Trial |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with Cystic Fibrosis who are Pa negative or concurrently enrolled in the EPIC Clinical Trial
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Time frame: 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.
Time frame: over the two-to-five-year observational period
7 secondary outcomes
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.
Time frame: over the two-to-five year observational period
To describe temporal changes in anti-pseudomonal serology and airway microbiology.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: over the two-to-five year observational period
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2019.
- Derived from this study: PubMed 22554417Rosenfeld 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.
- Derived from this study: PubMed 20597081Rosenfeld 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.
- Derived from this study: PubMed 19470318Treggiari 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret Rosenfeld, MD, MPH principal investigator
Seattle Children's Hospital
Ronald L. Gibson, MD, PhD principal investigator
Seattle Children's Hospital
Wayne J. Morgan, MD principal investigator
University of Arizona Health Sciences Center
54 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by CF Therapeutics Development Network Coordinating Center at 54 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 54 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: over the two-to-five-year observational period). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00676169, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Feb 2019.