Cystic Fibrosis (CF)IMPROVEDNCT07303621
Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population
An observational study of Cystic Fibrosis (CF), run by Hospices Civils de Lyon at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Cystic Fibrosis (CF)
- How many people
- 150 participants
- Where
- 1 siteFrance
- Study dates
- 9 Feb 2026to 9 Aug 2027
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Study population is from a tertiary care hospital. Children enrolled in the study are followed in the referral center for pediatric cystic fibrosis.
Who may be able to join
- Children aged 2 to 17 years old
- Having Cystic Fibrosis
- Treated by Elexacaftor/Tezacaftor and Ivacaftor (Trikafta® or Kaftrio®)
Who cannot join
- Allergy to previous CFTR modulator association (Ivacaftor, lumacaftor)
- Pregnant women
- Patient already enrolled in another study with CYP3A4 inhibitor
- Pulmonary transplant recipient
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Femme Mère Enfant (HFME)
Bron, 69029
Philippe REIX, M.D., +33 4 27 85 54 70, philippe.reix@chu-lyon.fr
About this study
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.
- Conditions
- Cystic Fibrosis (CF)
- Being tested
- There is no intervention as this is a prospective pharmacokinetics study.
- Keywords
- Trikafta, Elexacaftor, Ivacaftor, Cystic Fibrosis, pharmacokinetics
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
There is no intervention as this is a prospective pharmacokinetics study.Other
There is no intervention as this is a prospective pharmacokinetics study.
| Study group | Role | What they receive |
|---|---|---|
| Trikafta, Elexacaftor, Ivacaftor, Tezacaftor, Cystic Fibrosis, Population pharmacokinetics | Not described Receives: Other: There is no intervention as this is a prospective pharmacokinetics study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Trough Concentration [Cmin] of Elexacaftor, Ivacaftor and Tezacaftor
Measure residual concentration of Elexacaftor, Ivacaftor and Tezacaftor
Measured: 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
Measured: 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
Measured: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing
Secondary outcomes (5)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Dec 2025
- Study start9 Feb 2026
- Primary completion9 Aug 2027 (estimated)
- Study completion9 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis (CF)Studies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07303621
- Sponsor's protocol ID
- 69HCL25_0708
- Other IDs
- 2025-A01805-44
- Acronym
- IMPROVED
- Status last verified
- Feb 2026
- First submitted
- 19 Nov 2025
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No