Clinical trial NCT01104402: Early Intervention in Cystic Fibrosis Exacerbation
Official title: Early Intervention in Cystic Fibrosis Exacerbation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Keywords: telemedicine
spirometer
CF
pulmonary
home monitoring
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
Collaborators: name: University of Washington
name: National Institutes of Health (NIH)
name: Cystic Fibrosis Foundation
name: National Heart, Lung, and Blood Institute (NHLBI)
First posted: 2010-04-15
Enrollment: 267
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

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 criteria: 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

Primary outcomes:
measure: Change in FEV1
timeFrame: 12 months
Secondary outcomes:
measure: Cystic Fibrosis Respiratory Symptom Diary (CFRSD)
timeFrame: 12 months
measure: Pulmonary Exacerbations
timeFrame: 12 months
measure: Change in Health Related Quality of Life Scores as Assessed by the Cystic Fibrosis Questionnaire Revised (CFQ-R) (Respiratory Subscale Only(
timeFrame: Change from baseline to 12 months
measure: Treatment Burden
timeFrame: Change from baseline to 12 months
measure: Change in Prevalence of Resistant Species of Bacteria
timeFrame: 12 months
measure: Serious Adverse Events (SAE)
timeFrame: 12 months
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University CF Clinic
state: Maryland
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Johns Hopkins University
name: Noah Lechtzin, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Washington
name: Christopher Goss, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: VanDevanter EJ, Heltshe SL, Skalland M, Lechtzin N, Nichols D, Goss CH. The effect of oral and intravenous antimicrobials on pulmonary exacerbation recovery in cystic fibrosis. J Cyst Fibros. 2021 Nov;20(6):932-936. doi: 10.1016/j.jcf.2021.02.012. Epub 2021 Mar 5.
citation: Lechtzin N, Mayer-Hamblett N, West NE, Allgood S, Wilhelm E, Khan U, Aitken ML, Ramsey BW, Boyle MP, Mogayzel PJ Jr, Gibson RL, Orenstein D, Milla C, Clancy JP, Antony V, Goss CH; eICE Study Team. Home Monitoring of Patients with Cystic Fibrosis to Identify and Treat Acute Pulmonary Exacerbations. eICE Study Results. Am J Respir Crit Care Med. 2017 Nov 1;196(9):1144-1151. doi: 10.1164/rccm.201610-2172OC.