Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: There is no intervention as this is a prospective pharmacokinetics study..
- Who it may be relevant to
- Registry conditions: Cystic Fibrosis (CF). Basic parameters: 2 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
Cystic fibrosis is a rare, progressive genetic disease caused by a mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. Respiratory and nutritional effects are crucial to patients' prognosis. Since the early years of 2010, etiological treatment has been based on the use of CFTRm (CFTR modulator), which aim to restore the function of the mutated protein. Initially used as monotherapy and targeting a limited number of patients, CFTRm has gradually been extended to a larger number of patients, to the point where it now concerns 9 out of 10 patients, through the use of triple therapy with Elexacaftor-Tezacaftor-Ivacaftro (ETI) or Kaftrio(R). The efficacy of triple therapy is spectacular, revolutionizing the prognosis of the disease. However, the potential for neuropsychological side-effects (20-50% depending on age, but more frequent in young children under 5) and hepatic side-effects (hepatic cytolysis) must be taken into account. A better understanding of pharmacokinetic variability in children, as well as the relationship between exposure to therapeutic effects and adverse reactions, is therefore particularly important. The aim of this study is to measure the association between the pharmacokinetic parameters of Elexacaftor, Tezacaftor and Ivacaftor (plasma clearance and volume of distribution) and therapeutic or adverse effects in pediatric patients with cystic fibrosis treated with the combination.
Interventions
- Other There is no intervention as this is a prospective pharmacokinetics study.
There is no intervention as this is a prospective pharmacokinetics study.
Primary outcome measures
- Trough Concentration [Cmin] of Elexacaftor, Ivacaftor and Tezacaftor [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Maximum Plasma Concentration [Cmax] [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Area Under the Concentration Time Curve between two administrations of Elexacaftor, Ivacaftor and Tezacaftor [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
Secondary outcome measures (5)
- Number (Proportion) of Subjects with adverse events [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Relationship between Pharmacokinetics and Cystic Fibrosis mutational status and Adverse Event [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Relationship between Pharmacokinetics/Toxixodynamic and Cystic Fibrosis mutational status [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Number of Participants with Clinically Significant Changes in Clinical Laboratory Evaluations [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
- Area Under the Effect Time curve (AUEC) of Sweat Chloride [Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
Eligibility criteria
Inclusion criteria
- Children aged 2 to 17 years old
- Having Cystic Fibrosis
- Treated by Elexacaftor/Tezacaftor and Ivacaftor (Trikafta® or Kaftrio®)
Exclusion criteria
- Allergy to previous CFTR modulator association (Ivacaftor, lumacaftor)
- Pregnant women
- Patient already enrolled in another study with CYP3A4 inhibitor
- Pulmonary transplant recipient
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 1 center
- Hôpital Femme Mère Enfant (HFME) — Bron
Identifiers
NCT: NCT07303621 · 69HCL25_0708 · 2025-A01805-44