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NCT Number: NCT07809867

Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis

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.

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.

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Key information

Conditions

Cystic Fibrosis (CF) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • CF patients with at least one F508del mutation treated with the combination ETI 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.

Exclusion criteria

  • 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.

Treatment and study plan

Elexacaftor, Tezacaftor, Ivacaftor

Drug

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy

Ivacaftor (VX-770)

Drug

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy

Primary outcomes

  1. Plasma concentrations of elexacaftor, tezacaftor and ivacaftor at steady-state (Css)

    Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]

Secondary outcomes

  1. Population pharmacokinetics (popPK) analysis of elexacaftor, tezacaftor and ivacaftor at steady state

    Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]

  2. Harvest of every AE/Adverse Drug Reaction (ADR)

    Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]

  3. Correlation between pharmacokinetics of elexacaftor, tezacaftor and ivacaftor and number of Adverse Event(s)

    Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]

  4. 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

    Time frame: [Visit 1 - Day 0] [Visit 2 - Day 90±5] [Visit 3 - Day 180±5]

Sponsors and collaborators

Lead sponsor

Istituto Giannina Gaslini

Other

Registry information

Official study title

A Low-intervention Prospective-retrospective Study to Evaluate the Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Combination in a Cystic Fibrosis Population

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Sep 9, 2026
Registry last updated
Sep 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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