Assessing lung function in children with respiratory diseases

Measures of Respiratory Health

This study is testing a new way to measure lung function in children with cystic fibrosis and other breathing problems to see if it can help catch lung issues early, and healthy kids will also take part to help compare the results.

Quick facts

• Study type: Observational
• Enrollment: 300 (estimated)
• Ages: 30 Months to 30 Years
• Sex: All
• Sponsor: The Hospital for Sick Children (Academic / other)
• Locations: 1 site (Toronto, Ontario)
• Trial ID: NCT02657837 on ClinicalTrials.gov

What this trial studies

This observational study evaluates lung function in pediatric patients with cystic fibrosis and other respiratory diseases using the Lung Clearance Index (LCI) measured by multiple breath washout (MBW). The study aims to identify early signs of lung disease that may not yet present clinical symptoms, facilitating timely interventions. Healthy controls of various ages will also participate to provide reference data for interpreting LCI results in affected patients. The study is conducted at The Hospital for Sick Children in Toronto, Ontario, utilizing a device that is not yet approved by Health Canada for routine use.

Who should consider this trial

Good fit: Ideal candidates include children aged 2.5 to 18 years with a diagnosis of cystic fibrosis or other diagnosed lung diseases.

Not a fit: Patients requiring supplementary oxygen to maintain oxygen saturation above 95% or those with physical findings compromising safety may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to earlier detection and intervention for lung diseases in children, potentially improving long-term health outcomes.

How similar studies have performed: Other studies have shown promise in using similar approaches to assess lung function, but this specific methodology is still under evaluation in Canada.

Eligibility criteria

Participants with CF

Inclusion criteria:

1. 2.5 - 18 years of age at enrolment
2. Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF phenotype or positive CF newborn screen AND one or more of the following criteria:

   1. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis (QPIT)
   2. A documented genotype with two disease-causing mutations in the CFTR gene
3. Informed consent by participant, parent, or legal guardian
4. Ability to perform technically acceptable MBW measurements

Exclusion criteria:

1. Physical findings at screening that would compromise the safety of the participant as judged by the patient's most responsible physician
2. Requirement of supplementary oxygen to maintain an oxygen saturation above 95%

Participants with other respiratory disease

Inclusion criteria:

1. MD diagnosed lung disease and/or attending the Pulmonary Function Laboratory
2. 2.5 - 18 years of age at enrollment
3. Informed consent by participant, parent, or legal guardian
4. Ability to perform technically acceptable MBW measurements

Exclusion criteria:

1. Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
2. Requirement of supplementary oxygen to maintain an oxygen saturation above 95%

Healthy Participants

Inclusion criteria:

1. 2.5 - 30 years of age at enrollment
2. Informed consent by participant, parent, or legal guardian
3. Ability to perform technically acceptable MBW measurements

Exclusion criteria:

1. Physical findings at screening that would compromise the safety of the participant or the quality of the study data.
2. Evidence of a chronic disease process such as lung disease

Where this trial is running

Toronto, Ontario

• The Hospital for Sick Children — Toronto, Ontario, Canada (Recruiting)

Study contacts

• Principal investigator: Felix Ratjen, MD PhD — The Hospital for Sick Children
• Study coordinator: Felix Ratjen, MD PhD

Conditions Cystic Fibrosis; Asthma; Sickle Cell Anemia; Bronchiolitis Obliterans; CF; Multiple Breath Washout; Lung Clearance Index; Pediatrics