NCT07303621 Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
| NCT ID | NCT07303621 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Hospices Civils de Lyon |
| Condition | Cystic Fibrosis (CF) |
| Study Type | OBSERVATIONAL |
| Enrollment | 150 participants |
| Start Date | 2026-02-09 |
| Primary Completion | 2027-08-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 150 participants in total. It began in 2026-02-09 with a primary completion date of 2027-08-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07303621 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 17 Years, studying Cystic Fibrosis (CF). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07303621 currently recruiting?
Yes, NCT07303621 is actively recruiting participants. Contact the research team at romain.garreau@chu-lyon.fr for enrollment information.
Where is the NCT07303621 trial being conducted?
This trial is being conducted at Bron, France.
Who is sponsoring the NCT07303621 clinical trial?
NCT07303621 is sponsored by Hospices Civils de Lyon. The trial plans to enroll 150 participants.