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Идёт набор NCT07303621

Population Pharmacokinetics of Elexacaftor-tezacaftor-ivacaftor in a Paediatric Population

— Наблюдательное Cystic Fibrosis (CF)

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: There is no intervention as this is a prospective pharmacokinetics study..
Кому может быть актуально
Состояния в реестре: Cystic Fibrosis (CF). Базовые параметры: 2 лет — 17 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Франция
Следующий шаг
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Обзор

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.

Вмешательства

  • Другое There is no intervention as this is a prospective pharmacokinetics study.
    There is no intervention as this is a prospective pharmacokinetics study.

Первичные конечные точки

  • Trough Concentration [Cmin] of Elexacaftor, Ivacaftor and Tezacaftor [Срок оценки: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
  • Maximum Plasma Concentration [Cmax] [Срок оценки: 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 [Срок оценки: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]
Вторичные конечные точки (5)
  • Number (Proportion) of Subjects with adverse events [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: 5 minutes pre-dosing 3 to 4 hours post-dosing 6 to 7 hours post-dosing]

Критерии участия

Критерии включения

  • Children aged 2 to 17 years old
  • Having Cystic Fibrosis
  • Treated by Elexacaftor/Tezacaftor and Ivacaftor (Trikafta® or Kaftrio®)

Критерии исключения

  • Allergy to previous CFTR modulator association (Ivacaftor, lumacaftor)
  • Pregnant women
  • Patient already enrolled in another study with CYP3A4 inhibitor
  • Pulmonary transplant recipient

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Франция · 1 центр
  • Hôpital Femme Mère Enfant (HFME) — Bron

Идентификаторы

NCT: NCT07303621 · 69HCL25_0708 · 2025-A01805-44

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗