Early Intervention in Cystic Fibrosis Exacerbation

NCT01104402 | Completed Results DMC

• 3 other identifiers: NL001LECHTZ10A0R01HL103965
• Study Type: interventional
• Target: 267
• Locations: 1 country
• Sites: 3

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.

Conditions

Cystic Fibrosis

Keywords

telemedicine; spirometer; CF; pulmonary; home monitoring

Outcome Measures

Primary Outcomes (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

  12 months

Secondary Outcomes (6)

• Cystic Fibrosis Respiratory Symptom Diary (CFRSD)

  12 months

• Pulmonary Exacerbations

  12 months

• Change in Health Related Quality of Life Scores as Assessed by the Cystic Fibrosis Questionnaire Revised (CFQ-R) (Respiratory Subscale Only(

  Change from baseline to 12 months

• Treatment Burden

  Change from baseline to 12 months

• Change in Prevalence of Resistant Species of Bacteria

  12 months

Study Arms (2)

Standard Care

NO INTERVENTION

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

Home monitoring

ACTIVE COMPARATOR

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

Device: Home lung function and symptom monitoring

Interventions

Home lung function and symptom monitoring DEVICE

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

Also known as: Jaeger AM2 monitor

Home monitoring

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Johns Hopkins University: lead
• University of Washington: collaborator
• National Institutes of Health (NIH): collaborator
• Cystic Fibrosis Foundation: collaborator
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (3)

Johns Hopkins University CF Clinic

Baltimore, Maryland, 21205, United States

Location

University of Washington

Seattle, Washington, 98104-2499, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105-5371, United States

Location

Related Publications (2)

• 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.

  PMID: 33685776 DERIVED

• 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.

  PMID: 28608719 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Dr. Noah Lechtzin & Dr. Christopher Goss
• Organization: Johns Hopkins University & University of Washington

nlechtz@jhmi.edu

410-502-7044

Study Officials

• Noah Lechtzin, MD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

• Christopher Goss, MD

  University of Washington

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2010
• First Posted: April 15, 2010
• Study Start: October 1, 2011
• Primary Completion: August 1, 2015
• Study Completion: September 1, 2015
• Last Updated: October 23, 2017
• Results First Posted: October 23, 2017

Record last verified: 2017-09

Locations

US (3)