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AzTrials

Cystic FibrosiseICENCT01104402

Early Intervention in Cystic Fibrosis Exacerbation

A study testing Home lung function and symptom monitoring in people with Cystic Fibrosis, run by Johns Hopkins University at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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. Read more about this study

Who is it for?
Ages 14 years and older
Conditions the study is for
Cystic Fibrosis
What is being tested?
Home lung function and symptom monitoring
Type of treatment
Medical device
Could I receive a placebo?
No, everyone receives an active treatment
How many people
267 participants
Where
3 sitesUnited States
Study dates
Oct 2011to Sep 2015
Phase
Interventional
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 14 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Johns Hopkins University CF Clinic

    Baltimore, Maryland, 21205

  • University of Washington

    Seattle, Washington, 98104-2499

  • Seattle Children's Hospital

    Seattle, Washington, 98105-5371

Other trials in United States

About this study

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.

Read the 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.

Conditions
Cystic Fibrosis
Being tested
Home lung function and symptom monitoring
Keywords
telemedicine, spirometer, CF, pulmonary, home monitoring
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Home lung function and symptom monitoringDevice

    Also called Jaeger AM2 monitor

    subjects in the intervention arm will measure spirometry and CF symptoms with the use of a handheld device.

Study groups in NCT01104402
Study groupRoleWhat they receive
Standard CareNo interventionSubjects will receive education about signs and symptoms indicative of worsening CF.
Home monitoringActive comparatorSubjects will be randomized to monitor home spirometry and symptoms using a handheld device.
Receives: Device: Home lung function and symptom monitoring

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 12 months

Secondary outcomes (6)

  1. 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.

    Measured: 12 months

  2. Pulmonary Exacerbations

    Percentage of participants who experienced at least one acute pulmonary exacerbation

    Measured: 12 months

  3. 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.

    Measured: Change from baseline to 12 months

  4. 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.

    Measured: Change from baseline to 12 months

  5. 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.

    Measured: 12 months

  6. 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.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Apr 2010 (estimated)
  2. Study startOct 2011
  3. Primary completionAug 2015
  4. Study completionSep 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
267 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01104402
Sponsor's protocol ID
NL001
Other IDs
LECHTZ10A0, R01HL103965
Acronym
eICE
Status last verified
Sep 2017
First submitted
12 Apr 2010
Last updated
23 Oct 2017
Data monitoring committee
Yes

Publications

  • Colegate SP, Manning ER, Vancil A, Wang Z, Rao M, Gecili E, Palipana A, Ryan P, Szczesniak RD, Brokamp C. Acute exposure to fine ambient particulate matter and pulmonary exacerbations in cystic fibrosis: A case-crossover study. Atmos Environ X. 2026 Jan;29:100414. doi: 10.1016/j.aeaoa.2026.100414. Epub 2026 Feb 1.

    PubMed 42051905

  • 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.

    PubMed 33685776

  • 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.

    PubMed 28608719

Questions about this trial

What is this clinical trial studying?

A study testing Home lung function and symptom monitoring in people with Cystic Fibrosis, run by Johns Hopkins University at 3 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 6 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 23 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 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?

Yes. Results are posted in the registry (first posted 23 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change in FEV1 (time frame: 12 months). 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 NCT01104402, 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 23 Oct 2017.

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