How the body handles elexacaftor-tezacaftor-ivacaftor in children with cystic fibrosis
Studies how children with cystic fibrosis process elexacaftor-tezacaftor-ivacaftor and links this to effects.
Plain-language title generated from the registry record
Registry title: Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
Key facts
- Where
- Bron, FranceSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 17 years old?
Source: NCT07303621, who can take part
Where in France
- Hôpital Femme Mère Enfant (HFME)Bron, FranceTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Hospices Civils de Lyon
- Trial code
- NCT07303621
- Record updated
- 20 February 2026
- Original description
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).Read full original descriptionShow less
Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
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.
Inclusion: Children aged 2 to 17 years old; Having Cystic Fibrosis; Treated by Elexacaftor/Tezacaftor and Ivacaftor (Trikafta® or Kaftrio®).
Exclusion: Allergy to previous CFTR modulator association (Ivacaftor, lumacaftor); Patient already enrolled in another study with CYP3A4 inhibitor; Pulmonary transplant recipient; Pregnant women.