Early Intervention in Cystic Fibrosis Exacerbation

NCT ID: NCT01104402

Last Updated: 2017-10-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 267 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-10-31
• Study Completion Date: 2015-09-30

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.

Conditions

Cystic Fibrosis

Keywords

telemedicine; spirometer; CF; pulmonary; home monitoring

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Standard Care

Subjects will receive education about signs and symptoms indicative of worsening CF.

Group Type: NO_INTERVENTION

No interventions assigned to this group

Home monitoring

Subjects will be randomized to monitor home spirometry and symptoms using a handheld device.

Group Type: ACTIVE_COMPARATOR

Home lung function and symptom monitoring

Intervention Type: DEVICE

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

Interventions

Home lung function and symptom monitoring

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

Intervention Type: DEVICE

Other Intervention Names

Jaeger AM2 monitor

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

• Minimum Eligible Age: 14 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Washington

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Noah Lechtzin, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Christopher Goss, MD

Role: PRINCIPAL_INVESTIGATOR

University of Washington

Locations

Johns Hopkins University CF Clinic

Baltimore, Maryland, United States

University of Washington

Seattle, Washington, United States

Seattle Children's Hospital

Seattle, Washington, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 33685776 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 28608719 (View on PubMed)

Other Identifiers

LECHTZ10A0

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

R01HL103965

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NL001

Identifier Type: -

Identifier Source: org_study_id