Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.
Study design (if review, criteria of inclusion for studies)
Controlled: Yes.1 Randomised: Yes.2 Open: Yes.3 Single blind: No.4 Double blind: No.5 Parallel group: No.6 Cross over: No
Participants
Children and adolescents with CF and Aspergillus infection. Principal inclusion criteria: Inclusion criteria for the screening phase: 1. Diagnosed with cystic fibrosis (genetic diagnosis and/or abnormal sweat test and clinical phenotype of lung disease) - 2. Age ⥠8 yrs and < 18 yrs - 3. Body weight ⥠20 kg - 4. Presence of Aspergillus infection as defined for this study - 5. Clinically stable condition without a significant change in lung function (FEV1 +/- 10%) or significant worsening of respiratory symptoms over the previous month - 6. Able to perform lung function test (FEV1%) - 7. Able to produce a sputum sample (spontaneous or induced sputum) - 8. Informed Consent given - 9. If female and of childbearing age must be using highly effective contraception (and must agree to continue for 7 days after the last dose of investigational medicinal product [IMP]) [1[
Interventions
Paediatric posaconazole dosing regimen
Outcome measures
Primary end point(s): - For the dosing algorithm: The number of children and adolescents with CF and Aspergillus infection aged 8 to 17 years reaching the pre-defined area under the concentration time curve (AUC) of posaconazole at the first assessment (between day 5 and 10) based on the adult reference concentrations for treatment of susceptible pathogens.