cystic fibrosis mucoviscidosis
Conditions
Interventions
None listed
Sponsors
Erasmus MC, Universitair Medisch Centrum Rotterdam
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: In order to be eligible to participate in this study, a subject must meet all of the following criteria: Diagnosed with cystic fibrosis (CF), either by abnormal sweat test and/or confirmed with 2 CF causing mutations found by genetic analysis, either from heel-prick screening or diagnosed later in life. Furthermore, they have to be older than 6 years (i.e. children and adults). Age appropriate written informed consent is required. In addition, patients need to meet the criteria of one of the following study groups: Group 1: CF patients, whom are eligible to start elexacaftor/tezacaftor/ivacaftor treatment or who are already using it. Group 2: CF patients, whom are not eligible to receive elexacaftor/tezacaftor/ivacaftor treatment. This group will function as controls.
Exclusion criteria
Exclusion criteria: - Patients who cannot follow instructions
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary endpoint is the comparison of VOCs, measured by GC-MS, during ETI treatment compared to control group over time during 3 different study visits. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints: - The correlation of VOCs by GC-MS breath profiles/VOCs, measured by eNose, inflammatory markers in induced sputum (IL-8, free neutrophilic elastase (NE), calprotectin and myeloperoxidase, plus a predetermined cytokine panel), blood (IL-18, IL-1β, TNF, hsCRP, sCD14, calprotectin, HGMB-1, amyloid and miRNA), urine and, lung function, quality of life and symptom scores at baseline (if available) and overtime during 3 consecutive study visits. - Change of VOCs by GC-MS and eNose from baseline till 3 months of ETI treatment. | — |
Countries
Netherlands
Outcome results
None listed