A New Posaconazole Dosing Regimen for Paediatric Patients With Cystic Fibrosis and Aspergillus Infection

Summary

This study will provide: (1) new insights in the prevalence of Aspergillus infection in children and adolescents with CF aged 8-17 yrs; (2) an in silico modelled dose of posaconazole for children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (3) an intensive sampling PK study to define the optimal dose in a limited number of children and adolescents with CF and Aspergillus infection aged 8-17 yrs; (4) a prospective clinical validation to reduce the residual variability and to allow investigation into PK-PD; and (5) an efficacy evaluation of this dosing regimen to treat Aspergillus infection in children and adolescents with CF to inform future primary efficacy trials.

Conditions

• Cystic Fibrosis
• Aspergillosis

Interventions

DRUG

Posaconazole 100 MG [Noxafil]

Posaconazole is a lipophilic, highly permeable triazole, which is practically insoluble in water. Posaconazole inhibits the enzyme CYP51a (also known as Erg11p or lanosterol demethylase). This enzyme is required for catalysation of an essential step in biosynthesis of ergosterol in filamentous fungi and yeasts. Posaconazole is favoured over itraconazole and voriconazole with respect to palatability, tolerability and toxicity profile

DRUG

Posaconazole 40 MG/ML

Posaconazole is a lipophilic, highly permeable triazole, which is practically insoluble in water. Posaconazole inhibits the enzyme CYP51a (also known as Erg11p or lanosterol demethylase). This enzyme is required for catalysation of an essential step in biosynthesis of ergosterol in filamentous fungi and yeasts. Posaconazole is favoured over itraconazole and voriconazole with respect to palatability, tolerability and toxicity profile

Sponsors & Collaborators

• University of Exeter

  collaborator OTHER

• Radboud University Medical Center

  collaborator OTHER

• Consorzio per Valutazioni Biologiche e Farmacologiche

  collaborator OTHER

• Bambino Gesù Hospital and Research Institute

  lead OTHER

Principal Investigators

• Adilia Warris, Prof · University of Exeter

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

8 Years

Max Age

17 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2021-04-22

Primary Completion

2023-04-30

Completion

2023-11-30

Countries

• Czechia
• France
• Germany
• Greece
• Ireland
• Italy
• Netherlands
• Portugal
• Spain
• Switzerland
• United Kingdom

Study Locations