Early Intervention in Cystic Fibrosis Exacerbation

Cystic Fibrosis Clinical Trial

— eICE  

Official title:

Early Intervention in Cystic Fibrosis Exacerbation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01104402
• Other study ID #: NL001
• Secondary ID: LECHTZ10A0R01HL1
• Status: Completed
• Phase: N/A
• First received: April 12, 2010
• Last updated: September 22, 2017
• Start date: October 2011
• Est. completion date: September 2015

Study information

• Verified date: September 2017
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Individuals with cystic fibrosis (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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 267
• Est. completion date: September 2015
• Est. primary completion date: August 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

Inclusion Criteria:

• CF diagnosis confirmed with sweat test, abnormal nasal potential difference and/or genetic testing

• Age 14 and older

• Able to perform spirometry

• Clinically stable without antibiotic treatment for a pulmonary exacerbation in the two weeks prior to the screening visit

• Forced expiratory volume in the first second (FEV1) greater than 25% of predicted at screening

Exclusion Criteria:

• History of solid organ transplant

• Participation in any interventional trial within the last 30 days

• Inability to speak and read the English language well enough to complete questionnaires

• Colonization with Burkholderia cepacia genomovar III within the last 24 months

• Currently receiving antimicrobial treatment specifically used to treat active non-tuberculosis mycobacterium

• Confirmed diagnosis of allergic bronchopulmonary aspergillosis (ABPA) as defined by the Cystic Fibrosis Foundation (CFF) guidance document that is being actively treated

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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.

Locations

Country	Name	City	State
United States	Johns Hopkins University CF Clinic	Baltimore	Maryland
United States	Seattle Children's Hospital	Seattle	Washington
United States	University of Washington	Seattle	Washington

Sponsors (5)

Lead Sponsor	Collaborator
Johns Hopkins University	Cystic Fibrosis Foundation Therapeutics, National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), University of Washington

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in FEV1	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	12 months
Secondary	Cystic Fibrosis Respiratory Symptom Diary (CFRSD)	Change in CF respiratory symptoms as measured by the CFRSD. The CFRSD consists of 8 items which quantify symptom severity for the previous 24 hours to capture the magnitude of symptoms in stable CF, during medically treated CF exacerbations, and during recovery from an exacerbation. The CFRSD also includes emotional and activity impacts. Emotional impacts include frustration, sadness/depression, irritability, worry, and difficulty sleeping. Activity impacts include time spent sitting or lying down, reduction of usual activities, and missing school or work. 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. The range of scores is 8 to 40 with higher scores indicating more severe symptoms.	12 months
Secondary	Pulmonary Exacerbations	Percentage of participants who experienced at least one acute pulmonary exacerbation	12 months
Secondary	Change in Health Related Quality of Life Scores as Assessed by the Cystic Fibrosis Questionnaire Revised (CFQ-R) (Respiratory Subscale Only(	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. The CFQ-R measures functioning in a variety of domains, including Physical Functioning, Vitality, Health Perceptions, Respiratory Symptoms, Treatment Burden, Role Functioning, Emotional Functioning, and Social Functioning. Only the respiratory subscale of the the CFQ-R was evaluated. This ranges from 0 to 100 with higher scores indicating better respiratory quality of life. A negative number indicates a decrease in respiratory quality of life.	Change from baseline to 12 months
Secondary	Treatment Burden	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. Scores range from 0-100 with higher scores indicating less treatment burden.	Change from baseline to 12 months
Secondary	Change in Prevalence of Resistant Species of Bacteria	Change in prevalence of resistant species of bacteria (Methicillin Resistant S. aureus, Pseudomonas aeruginosa, Burkolderia cepacia, Stenotrophomona maltophilia, Achromobacterxylosoxidans) in sputum between baseline and final visit (Visit 5 or early withdrawal) will be summarized by treatment group.	12 months
Secondary	Serious Adverse Events (SAE)	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.	12 months