How Trikafta (elexacaftor‑tezacaftor‑ivacaftor) is handled by children’s bodies with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This prospective observational study will collect timed blood samples from children aged 2–17 who are already taking elexacaftor‑tezacaftor‑ivacaftor to measure drug concentrations and build a population pharmacokinetic model. Researchers will examine how factors such as age, weight, liver function, and concomitant medications influence drug exposure. No changes to prescribed therapy will be made and standard safety monitoring (including liver tests and clinical follow-up) will be performed. The aim is to generate pediatric-specific PK data to inform dosing and monitoring strategies and help reduce adverse effects.

Why it matters

Who can join: the full rules

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

Where it's running

• Hôpital Femme Mère Enfant (HFME)
  Bron, France Recruiting

Study contacts

• Study coordinator: Romain GARREAU, PharmD.
• Email: romain.garreau@chu-lyon.fr
• Phone: +33 4 72 07 19 28

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.