Pharmacokinetics of tezacaftor-ivacaftor (Symkevi) in children with cystic fibrosis
- Conditions
- Cystic Fibrosis
- Registration Number
- NL-OMON20087
- Lead Sponsor
- one
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 30
Use a combination therapy of tezacaftor-ivacaftor for a minimum period of 8 days in regular care or compassionate use
- CF patients aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G,
- 711+3A?G, S945L, S977F, R1070W, D1152H, 2789+5G?A, 3272-26A?G, and 3849+10kbC?T.
- 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.
- 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
Study & Design
- Study Type
- Observational non invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To assess the exposure (AUC, Cmax) of tezacaftor-ivacaftor in a real life clinical setting in paediatric CF patients
- Secondary Outcome Measures
Name Time Method 1) To evaluate the relationship between covariates and PK parameters in order to explain inter-patient variability<br>2) To evaluate the relationship between AUC and through levels<br>3) To compare drug exposure in children of different age groups and compare with that in adults<br>4) To explore if there is a correlation between drug concentrations and clinical outcome measures (efficacy like exacerbation frequency, increase in weight, lung