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

NCT ID: NCT07303621

Last Updated: 2025-12-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

150 participants

Study Classification

OBSERVATIONAL

Study Start Date

2026-01-01

Study Completion Date

2027-02-01

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis (CF)

Keywords

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Trikafta Elexacaftor Ivacaftor Cystic Fibrosis pharmacokinetics

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Trikafta, Elexacaftor, Ivacaftor, Tezacaftor, Cystic Fibrosis, Population pharmacokinetics

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

Intervention Type OTHER

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

Interventions

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

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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Hôpital Femme Mère Enfant (HFME)

Bron, , France

Site Status

Countries

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France

Central Contacts

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Romain GARREAU, PharmD.

Role: CONTACT

Phone: +33 4 72 07 19 28

Email: [email protected]

Philippe REIX, M.D., Ph.D

Role: CONTACT

Phone: +33 4 27 85 54 70

Email: [email protected]

Facility Contacts

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Philippe REIX, M.D.

Role: primary

Other Identifiers

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2025-A01805-44

Identifier Type: OTHER

Identifier Source: secondary_id

69HCL25_0708

Identifier Type: -

Identifier Source: org_study_id