Cystic Fibrosis (CF)
Conditions
Interventions
This study will be an observational study. We will observate the intervention of starting with a treatment with Ivacaftor.
Sponsors
University Medical Center Utrecht (UMCU)
Eligibility
Inclusion criteria
Inclusion criteria: • CFTR genotype compound/ S1251N • Already had a rectal biopsy to produce an organoid • Start a treatment with Ivacaftor • Male and female patients, aged 6 years or older on the date of informed consent or; • Signed informed consent form (IC).
Exclusion criteria
Exclusion criteria: • Use of curcumin and or genistein at start or within two weeks prior to start of the study; • Inability to follow instructions of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Sweat chloride concentration (SCC) before and after treatment with Ivacaftor. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Pulmonary function (%FEV1) and airway resistance (Rint and bodybox). • BMI (=weight (in Kg)/Length2 (in cm)); • Quality of life (measured with CFQ-questionnaire) • Bile salt measurements in plasma and the feces. • Elastase measurements in the feces • Correlation between individual Ivacaftor induced CFTR function in vitro (organoid-based measurements) and the in vivo treatment effect; • The CFTR stimulating ability of the concentration of Ivacaftor in the patient’s blood samples, examined by in vitro testing (in the organoid model). We will also determine the plasma levels of Ivacaftor. | — |
Contacts
Public ContactS. Michel
Wilhelmina Kinderziekenhuis Huispostnummer KH 01.419.0 Postbus 85090
Outcome results
None listed