Early Intervention in Cystic Fibrosis Exacerbation (eICE)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
University of Washington
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Noah Lechtzin, Johns Hopkins University
ClinicalTrials.gov Identifier:
NCT01104402
First received: April 12, 2010
Last updated: July 24, 2014
Last verified: July 2014
  Purpose

Individuals with CF develop chronic lung infections and suffer intermittent acute exacerbations of their lung disease. Most exacerbations are not treated until they cause increased symptoms, and patients seek medical attention. This proposal details a study of home lung function and symptom monitoring. Subjects will be randomly assigned to one of two groups: 1) home monitoring, in which spirometry and symptoms are recorded; or 2) standard care. The home monitoring data will be transmitted electronically to the study center. If spirometry or symptoms have deteriorated substantially, treatment for a CF pulmonary exacerbation will be initiated. It is anticipated that use of home monitoring will lead to earlier, more reliable recognition and treatment of exacerbations, which will translate into better lung health.


Condition Intervention Phase
Cystic Fibrosis
Device: Home lung function and symptom monitoring
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Early Intervention in Cystic Fibrosis Exacerbation

Resource links provided by NLM:


Further study details as provided by Johns Hopkins University:

Primary Outcome Measures:
  • Change in FEV1 [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    The primary outcome variable is FEV1 which will be obtained at quarterly study visits. The primary analysis will use a linear mixed effects model incorporating all FEV1 measurements to estimate the 52-week change in FEV1 and test for the differences between the two treatment groups (Early Intervention and Usual Care).


Secondary Outcome Measures:
  • Respiratory Symptom Scores (CFRSD) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Change in CF respiratory symptoms as measured by the CFRSD will be analyzed using a linear mixed effects model incorporating baseline randomization factors FEV1 (<50%, 50-75%, and >75% predicted) and age (14-18 & 19+), treatment group, time (in weeks) and the interaction between treatment and time.

  • Pulmonary Exacerbations [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Time to first acute protocol-defined pulmonary exacerbation and time from the start of acute pulmonary exacerbation to onset of subsequent pulmonary exacerbation will be analyzed using proportional hazards regression.

  • Change in Health Related Quality of Life (QOL)Scores [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Change in health related quality of life as measured by the Cystic Fibrosis Questionnaire revised (CFQ-R)will be analyzed using a linear mixed effects model incorporating baseline randomization factors FEV1 (<50%, 50-75%, and >75% predicted) and age (14-18 & 19+), treatment group, time (in weeks) and the interaction between treatment and time.

  • Treatment Burden [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    Change in treatment burden as measured by the Cystic Fibrosis Questionnaire revised (CFQ-R)will be analyzed using a linear mixed effects model incorporating baseline randomization factors FEV1 (<50%, 50-75%, and >75% predicted) and age (14-18 & 19+), treatment group, time (in weeks) and the interaction between treatment and time.

  • Change in prevalence of resistant species of bacteria [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    Change in prevalence of resistant species of bacteria (Methicillin Resistant S. aureus, Multiply Resistant P. aeruginosa, B. cepacia, S. maltophilia, A xylosoxidans) in sputum between baseline and final visit (Visit 5 or early withdrawal) will be summarized by treatment group.

  • Adverse Events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    Adverse event rates will be coded by body system and MedDRA classification term. Adverse events will be tabulated by treatment group and will include the number of subjects for whom the event occurred, the rate of occurrence, and the severity and relationship to study participation or study procedures.


Estimated Enrollment: 140
Study Start Date: October 2011
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: Standard Care
Subjects will receive education about signs and symptoms indicative of worsening CF.
Experimental: Home Monitoring
Subjects will be randomized to monitor home spirometry and symptoms using a handheld device.
Device: Home lung function and symptom monitoring
subjects in the intervention arm will measure spirometry and CF symptoms with the use of a handheld device.
Other Name: Jaeger AM2 monitor

Detailed Description:

Individuals with CF develop chronic lung infections and suffer intermittent exacerbations, which require intensive treatment with antibiotics. The most common and useful objective measure of CF lung disease is spirometry. Chronic treatment of CF lung disease requires airway clearance, mucolytics and antibiotics. These treatments have been quite successful and there is evidence that early, aggressive treatment of lung disease results in better outcomes. Unfortunately, most exacerbations are not treated until they cause pronounced deterioration in symptoms, which prompts patients to seek medical attention. Self-monitoring of clinical status has improved outcomes in many other disorders such as asthma, diabetes mellitus, and lung transplantation. This is an important, randomized trial of home lung function and symptom monitoring in CF. Subjects will be assigned to one of two groups: 1) Home monitoring, in which spirometry and symptoms are recorded daily; or 2) Standard Care. The home monitoring data will be transmitted electronically twice weekly to the study center, where the results will be reviewed. If spirometry or symptoms have deteriorated substantially below baseline, treatment for a CF pulmonary exacerbation will be initiated. It is anticipated that use of home monitoring will translate into better clinical outcomes. We will test the hypothesis that if pulmonary exacerbations are identified and treated earlier than the current standard of care, the progression of lung disease will be slowed.

  Eligibility

Ages Eligible for Study:   14 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. CF diagnosis confirmed with sweat test, abnormal nasal potential difference and/or genetic testing
  2. Age 14 and older
  3. Able to perform spirometry
  4. Clinically stable without antibiotic treatment for a pulmonary exacerbation in the two weeks prior to the screening visit
  5. Forced expiratory volume in the first second (FEV1) greater than 25% of predicted at screening

Exclusion Criteria:

  1. History of solid organ transplant
  2. Participation in any interventional trial within the last 30 days
  3. Inability to speak and read the English language well enough to complete questionnaires
  4. Colonization with Burkholderia cepacia genomovar III within the last 24 months
  5. Currently receiving antimicrobial treatment specifically used to treat active non-tuberculosis mycobacterium
  6. Confirmed diagnosis of allergic bronchopulmonary aspergillosis (ABPA) as defined by the CFF guidance document that is being actively treated
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01104402

Locations
United States, Maryland
Johns Hopkins University CF Clinic
Baltimore, Maryland, United States, 21205
United States, Washington
University of Washington
Seattle, Washington, United States, 98104-2499
Seattle Children's Hospital
Seattle, Washington, United States, 98105-5371
Sponsors and Collaborators
Johns Hopkins University
University of Washington
Cystic Fibrosis Foundation
Investigators
Principal Investigator: Noah Lechtzin, MD Johns Hopkins University
Principal Investigator: Christopher Goss, MD University of Washington
  More Information

No publications provided

Responsible Party: Noah Lechtzin, Assistant Professor of Medicine, Johns Hopkins University
ClinicalTrials.gov Identifier: NCT01104402     History of Changes
Other Study ID Numbers: NL001, LECHTZ10A0
Study First Received: April 12, 2010
Last Updated: July 24, 2014
Health Authority: United States: Institutional Review Board
United States: Data safety monitoring board

Keywords provided by Johns Hopkins University:
telemedicine
spirometer
CF
pulmonary
home monitoring

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on August 28, 2014