WP3 - Performing a single dose pharmacokinetic trial in children under six years of age (DEEP-1)

The selection of the dosing regimen and recommendations for dose adjustments across the various age groups in a paediatric indication may be based on PK and PK/PD bridging concepts. Therefore, the following objectives are addressed in the present WP:

•    to characterise the PK and the related PK/PD relationship of DFP following single oral dose in male and female paediatric patients with β-thalassemia major and other transfusion dependent-haemoglobinopathies aged up to 6 years
•    to evaluate the effect of demographic factors on the PK and the related PK/PD of DFP in children
•    to demonstrate the relevance of clinical trial simulations as tool for optimising the dosing regimen in children following the administration of DFP

WP Leader: Oscar della Pasqua (UL)
Involved Partners: CVBF, UL, AOC, MPHS, CU, ApoPharma, GIAMBRONE, AORC, AODP, PB, FGB