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

April 12, 2010
July 24, 2014
October 2011
July 2015   (final data collection date for primary outcome measure)
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).
Change in FEV1 [ Time Frame: 12 months ] [ Designated as safety issue: No ]
The primary outcome for the trial is the difference in change in FEV1 over 12 months between the home monitoring group and the CF Education group. This difference will be based on both the change in FEV1 from baseline to month 12 and on the FEV1 obtained as the slope of the best FEV1 measurements recorded in the pulmonary function laboratory at the quarterly visits.
Complete list of historical versions of study NCT01104402 on ClinicalTrials.gov Archive Site
  • 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.
  • Change in CF specific FEV1 percentile [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    change in CF-specific FEV1 percentile relative to the baseline value
  • Frequency of Severe Pulmonary exacerbations as a safety measure [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    We will compare the frequency of CF pulmonary exacerbations requiring IV antibiotics between the two study arms to determine if there is an undo risk of morbidity in one arm vs. the other.
  • Number of Clinic Visits [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    We will compare the number of clinic visits over 12 months in the two arms.
  • Quality of Life (QOL)Scores [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    We will compare QOL as mesured by the CFQ-R between study arms.
  • Comparing self reported and prescription refill data to determine treatment adherence [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Treatment adherence, both self reported and measured by pharmacy refill data will be compared between the two study arms over the 12 months of the study.
Not Provided
Not Provided
 
Early Intervention in Cystic Fibrosis Exacerbation
Early Intervention in Cystic Fibrosis Exacerbation

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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Cystic Fibrosis
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
  • 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.
    Intervention: Device: Home lung function and symptom monitoring
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
140
July 2016
July 2015   (final data collection date for primary outcome measure)

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
Both
14 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01104402
NL001, LECHTZ10A0
Yes
Noah Lechtzin, Johns Hopkins University
Johns Hopkins University
  • University of Washington
  • National Institutes of Health (NIH)
  • Cystic Fibrosis Foundation
Principal Investigator: Noah Lechtzin, MD Johns Hopkins University
Principal Investigator: Christopher Goss, MD University of Washington
Johns Hopkins University
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP