Cystic Fibrosis (CF)Phase 4NCT07809867
Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis
A Phase 4 study testing Elexacaftor, Tezacaftor, Ivacaftor and Ivacaftor (VX-770) in people with Cystic Fibrosis (CF), run by Istituto Giannina Gaslini at 1 site in Italy.
Trial summary
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics. A better understanding of the pharmacokinetic profiles of elexacaftor, tezacaftor, and ivacaftor is essential to support Therapeutic Drug Monitoring (TDM) and to guide dose… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis (CF)
- What is being tested?
- Elexacaftor, Tezacaftor, Ivacaftor and Ivacaftor (VX-770)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteItaly
- Study dates
- 18 Sep 2026to Apr 2028
- Phase
- Phase 4
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Cystic Fibrosis patients with at least one F508del mutation treated with the combination elexacaftor/tezacaftor/ivacaftor for a time sufficient to reach steady-state (8 days)
- males and females aged ≥6 years
- informed consent to participate in the study and to process the patient's personal data obtained prior to the collection of any study data.
Who cannot join
- evidence of inadequate compliance to treatment;
- pregnancy and/or breastfeeding;
- any conditions that may affect the ability to complete informed consent;
- any other severe systemic disorders that may compromise the PK parameters;
- use of any drug capable of moderately/strongly inhibiting or strongly inducing hepatic biotransformation (see section 4 for details);
- denial of the informed consent.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
IRCCS Istituto Giannina Gaslini
Genova
U.O.C. Servizio Sperimentazioni Cliniche Pediatriche, +39 010 5636 2221, sperimentazionicliniche@gaslini.org
About this study
Official title: A Low-intervention Prospective-retrospective Study to Evaluate the Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Combination in a Cystic Fibrosis Population
The goal of this clinical trial is to support the development of personalized therapy tailored to each patient's individual characteristics. A better understanding of the pharmacokinetic profiles of elexacaftor, tezacaftor, and ivacaftor is essential to support Therapeutic Drug Monitoring (TDM) and to guide dose adjustments when clinically indicated, without compromising therapeutic efficacy.
The study will include male and female patients aged ≥6 years with Cystic Fibrosis (CF) carrying at least one F508del mutation and receiving treatment with the elexacaftor/tezacaftor/ivacaftor combination for a sufficient period to achieve steady state.
Read more
The main objective of the study is to characterize the steady-state plasma concentration profiles of elexacaftor, tezacaftor, and ivacaftor in patients with Cystic Fibrosis treated with the elexacaftor/tezacaftor/ivacaftor combination.
Participants will continue their usual treatment with elexacaftor/tezacaftor/ivacaftor and ivacaftor according to the prescribed dosing schedule. During the study, they will attend regular clinical visits every 3 months for up to 12 months.
At these visits, blood samples will be collected as part of routine clinical care and used to measure the plasma concentrations of the study drugs. Additional blood samples may be collected after the morning dose to better characterize drug levels over time. In a subset of participants, additional blood samples may be collected at several time points during the day to further assess the pharmacokinetic profile of the drugs.
A single additional blood sample may be collected for pharmacogenetic analyses. Participants may also undergo a nasal brushing, as part of routine procedures, to obtain nasal epithelial cells for further analyses.
Clinical information, including routine laboratory tests, vital signs, treatment information and any adverse events, will be collected throughout the study. The results of the pharmacokinetic and other laboratory analyses will not be used to modify the participant's treatment, which will remain under the responsibility of the treating physician.
- Conditions
- Cystic Fibrosis (CF)
- Being tested
- Elexacaftor, Tezacaftor, Ivacaftor, Ivacaftor (VX-770)
- Keywords
- Cystic Fibrosis, Elexacaftor, Tezacaftor, Ivacaftor, Kaftrio, Kalydeco
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elexacaftor, Tezacaftor, IvacaftorDrug
Cystic Fibrosis Transmembrane Regulator (CFTR) modulator therapy
Ivacaftor (VX-770)Drug
Cystic Fibrosis Transmembrane Regulator (CFTR) modulator therapy
| Study group | Role | What they receive |
|---|---|---|
| Elexacaftor/Tezacaftor/Ivacaftor + Ivacaftor | Experimental | Not described Receives: Drug: Elexacaftor, Tezacaftor, Ivacaftor; Drug: Ivacaftor (VX-770) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma concentrations of elexacaftor, tezacaftor and ivacaftor at steady-state
Measured: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Secondary outcomes (4)
Population pharmacokinetics (popPK) analysis of elexacaftor, tezacaftor and ivacaftor at steady state
Measured: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Harvest of every Adverse Event (AE)/Adverse Drug Reaction (ADR)
Measured: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Correlation between pharmacokinetics of elexacaftor, tezacaftor and ivacaftor and number of Adverse Event (AE)
Measured: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Correlation between pharmacokinetics and elexacaftor, tezacaftor and ivacaftor efficacy in the 3 groups classifications (responders, non responders and unresolved responders) of elexacaftor, tezacaftor and ivacaftor
Measured: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2026
- Study start18 Sep 2026
- Primary completionApr 2028 (estimated)
- Study completionApr 2028 (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.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Istituto Giannina Gaslini
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07809867
- Sponsor's protocol ID
- PKCAFTORs
- Other IDs
- 2024-518785-28-00
- Status last verified
- Oct 2026
- First submitted
- 3 Sep 2026
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No