An observational study in Cystic Fibrosis (CF), sponsored by Hospices Civils de Lyon. Recruiting at 1 site in France. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-02-20.
Sponsored by Hospices Civils de Lyon · Observational
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 150 is above the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Hospices Civils de Lyon is the lead sponsor of 1,826 studies on the registry; 439 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Study population is from a tertiary care hospital. Children enrolled in the study are followed in the referral center for pediatric cystic fibrosis.
Exclusion Criteria:
Other: There is no intervention as this is a prospective pharmacokinetics study.
There is no intervention as this is a prospective pharmacokinetics study.
Trough Concentration [Cmin] of Elexacaftor, Ivacaftor and Tezacaftor
Measure residual concentration 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]
Measured Cmax of Elexacaftor, Ivacaftor and Tezacaftor
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
Area under the concentration time curve between two administrations of (AUC0-tz) of Elexacaftor, Ivacaftor and Tezacaftor
Time frame: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing
Number (Proportion) of Subjects with adverse events
Specific drug related adverse events such as hepatic impairment or neurocomportmental disorder will be monitored. All safety data will be analysed using descriptive statistics. Pharmacokinetics analysis will be used to monitor existing relationship between elexacaftor/tezacaftor and ivacaftor
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
Area Under the Effect Time curve (AUEC) of Lung Clearance Index 2.5
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
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Hospices Civils de Lyon