E84.0
Conditions
Interventions
Group 1: No control group, only interventional.
Children with CF treated with Lumacaftor/Ivacaftor for the first time are examined during their routine 3-monthly scheduled appointments in our cystic
Sponsors
Children's Hospital, University medical center Mainz
Eligibility
Sex/Gender
All
Age
2 Years to 12 Years
Inclusion criteria
Inclusion criteria: Age 2-12 Confirmed CF-diagnosis and mutation Planned to start medication with Lumacftor/Ivacaftor Written informed consent from parent or legal guardian
Exclusion criteria
Exclusion criteria: Comorbidity which exclude therapy with Lumacaftor/Ivacaftor (e.g liver or renal insufficiency) Clinically significant abnormality Respiratory infection at the start of treatment Pulmonary infection with resistant strain of bacteria
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess changes in diversity and composition of respiratory and intestinal microbiome. | — |
Secondary
| Measure | Time frame |
|---|---|
| To assess changes in Lung Clearance Index, FEV1 and FEV75-25, BMI, FFM, Immunoglobulin G and Il-8 in blood, neutrophil elastase and IL-8 in sputum, urine cathepsine and faecal calprotectin in stool. Changes in concentration of sweat chloride. Assessment of CF metabolome by mass spectroscopy for CF-specific biomarkers. | — |
Countries
Germany
Contacts
Public ContactKrystyna Poplawska
Zentrum für Kinder- und Jugendmedizin der Universitätsmedizin Mainz
Outcome results
None listed