How Trikafta (elexacaftor‑tezacaftor‑ivacaftor) is handled by children’s bodies with cystic fibrosis
Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
This project will test how Trikafta (elexacaftor‑tezacaftor‑ivacaftor) is absorbed, distributed, metabolized, and eliminated in children aged 2–17 with cystic fibrosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 150 (estimated) |
| Ages | 2 Years to 17 Years |
| Sex | All |
| Sponsor | Hospices Civils de Lyon Academic / other |
| Locations | 1 site (Bron) |
| Trial ID | NCT07303621 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Children aged 2–17 with cystic fibrosis who are already being treated with elexacaftor‑tezacaftor‑ivacaftor and can attend clinic visits at the study site are ideal candidates.
Not a fit: Infants under 2, adults, people not taking Trikafta/Kaftrio, patients with prior allergy to CFTR modulators, recent pulmonary transplant recipients, or pregnant people are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, the results could improve pediatric dosing and monitoring of Trikafta to reduce side effects and enhance treatment effectiveness for children with cystic fibrosis.
How similar studies have performed: Pharmacokinetics of ETI have been characterized in adults and some older pediatric groups, but prospective population PK data specifically covering 2–17‑year‑olds remain limited.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Bron
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.