cystische fibrose cystic fibrosis
Conditions
Interventions
None listed
Sponsors
Academisch Medisch Centrum
Eligibility
Age
2 Years to 17 Years
Inclusion criteria
Inclusion criteria: - Use a combination therapy of (elexacaftor-)tezacaftor-ivacaftor for a minimum period of 8 days in regular care or compassionate use - CF patients aged 6 years and older - Signed informed consent from the patient when >=16 years, from the patient and both parents for patients aged 12-15 years, from both parents aged 6-11 years
Exclusion criteria
Exclusion criteria: - History of poor compliance deemed by the physician - Concomitant use of drugs that have an inhibitory or inducing effect on the CYP3A4 enzyme metabolism 14 days before the blood collection, if the patient uses one or more of these medicines the blood collection of the upcoming visit will be skipped: o Inducers of CYP3A: rifampicin, rifabutin, phenobarbital, carbamazepine, phenytoin and St. John*s wort o Inhibitors of CYP3A: ketoconazole, itraconazole, posaconazole, voriconazole, telithromycin, clarithromycin, fluconazole, erythromycin and grapefruit juice - Patient or parent refusal
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is to determine the exposure (area under the curve (AUC) and maximum plasma concentration (Cmax)) of elexacaftor, tezacaftor and ivacaftor. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) To evaluate the relationship between covariates and PK parameters in order to explain inter-patient variability 2) To evaluate the relationship between AUC and through levels 3) To compare drug exposure in children of different age groups and compare with that in adults 4) To explore if there is a correlation between drug concentrations and clinical outcome measures (efficacy like exacerbation frequency, increase in weight, lung function parameters and safety like side effects. | — |
Countries
Netherlands
Outcome results
None listed