Pharmacokinetics of tezacaftor-ivacaftor (Symkevi) in children with cystic fibrosis

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON20087

Enrollment

Registered

2021-04-20

Start date

2021-05-01

Completion date

Unknown

Last updated

2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Interventions

The intervention consists of additional DBS blood sampling for PK analysis.

Eligibility

Inclusion criteria

Inclusion criteria: - 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.

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
To assess the exposure (AUC, Cmax) of tezacaftor-ivacaftor in a real life clinical setting in paediatric CF patients	—

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	—

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

—

Contacts

Public ContactSteffie Vonk

Amsterdam UMC - location AMC

s.e.vonk@amsterdamumc.nl020-7323475

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)