Tracking CF Lung Disease Through the Early Years: Utility of the LCI (LCITRACK)

This is a prospective observational study to follow a cohort of patients with Cystic Fibrosis and healthy controls for a period of two years. This study will include monitoring the subjects lung clearance index (by performing a breathing test called the multiple breath washout), as well as spirometry and their respiratory symptoms every three months as well as during a pulmonary exacerbation and after their recovery.

Study Overview

• Status: Completed
• Conditions: Observational
• Intervention / Treatment: Other: This is an observational study, no intervention used.

Detailed Description

This is a prospective observational cohort study. During the 3 year study period, each participant will be followed for a period of 2 years. The study will include quarterly measurements of the lung clearance index (LCI), as well as spirometry, for CF patients at their routine clinic visits. Parents of patients with CF will be asked to call the study nurse or clinical nurse if they experience a worsening of pulmonary symptoms and to come to the clinic for assessment of lung function. CF patients will then be assessed by a CF physician to assess whether they require antibiotic treatment based on a clinicians decisions to treat with antibiotics. The treatment decision will be left to the discretion of the patient's responsible physician, who will be blinded to the MBW results. All patients who meet the symptom based definition of a pulmonary exacerbation, regardless of treatment decision, will have MBW measured after 4 weeks. Following these visits at the time of an exacerbation, patients will have their MBW measured at their next clinic visit (usually within 3 months), and every 3 months thereafter until the end of the 2 year observation period, or the repeat onset of symptoms.This study will capture a maximum of two exacerbations per patient over the 2 year study period.

• Study Type: Observational
• Enrollment (Actual): 146

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G1X8
      • Hospital for Sick Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

There were 139 subjects recruited in the original preschool longitudinal study, and all participants from this original study will be invited to participate in this follow up study. We would like to recruit an additional 40 CF patients at the Hospital for Sick Children to increase our cohort size.

Description

Inclusion Criteria for Healthy Controls:

• Informed consent by patient or parent/legal guardian, verbal assent where appropriate
• Age 3 years to 16 years old
• Clinically stable with no signs of an acute exacerbation at enrollment visit
• Previously participated in a longitudinal MBW study at the Hospital for Sick Children and at the Riley Hospital for Children

Inclusion Criteria for CF Patients:

• Diagnosis of CF as defined by two or more clinical features of CF and a documented sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis test or a genotype showing two well characterized disease causing mutations
• Informed consent by patient or parent/legal guardian, verbal assent where appropriate
• Age 3 years to 16 years old
• Clinically stable with no signs of an acute exacerbation at enrollment visit

Exclusion Criteria:

• Previous organ transplantation
• Chronic lung disease not related to CF, such as asthma
• Use of intravenous antibiotics or other course of oral antibiotics (excluding maintenance treatment antibiotics) within 14 days of enrollment visit
• Physical findings at screening that would compromise the safety of the participant or the quality of the study data

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Healthy Controls; No intervention. Only monitoring LCI	Other: This is an observational study, no intervention used.; No intervention is being used in this study.
Cystic Fibrosis Patients; No intervention. Only monitoring LCI	Other: This is an observational study, no intervention used.; No intervention is being used in this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung Clearance Index	Outcome measure from the Multiple Breath Washout test.	an average of 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Spirometry	FEV1	an average of 2 years
Respiratory Cultures	Bacterial pathogens IL-8 and neutrophil elastase	an average of 2 years
Respiratory Symptoms	CFQ-R	an average of 2 years
Respiratory Symptoms	CFRSD-CRISS	an average of 2 years

Collaborators and Investigators

• Sponsor: Felix Ratjen
• Investigators: Principal Investigator: Felix Ratjen, MD, The Hospital for Sick Children

Study record dates

Study Major Dates

• Study Start (Actual): October 5, 2017
• Primary Completion (Actual): October 1, 2020
• Study Completion (Actual): September 1, 2021

Study Registration Dates

• First Submitted: April 27, 2017
• First Submitted That Met QC Criteria: May 1, 2017
• First Posted (Actual): May 3, 2017

Study Record Updates

• Last Update Posted (Actual): September 21, 2021
• Last Update Submitted That Met QC Criteria: September 20, 2021
• Last Verified: September 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases
• Other Study ID Numbers: 1000055762

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No