Pharmacokinetic Study of Deferiprone in Paediatric Patients
Status:
Completed
Trial end date:
2014-02-01
Target enrollment:
Participant gender:
Summary
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been
authorised in Europe in 1999 for the treatment of iron overload in patients with
beta-thalassaemia major when DFO is contraindicated or inadequate. Despite a wide experience
of DFP there are limited experimental data available on DFP in children and no
pharmacokinetic data in children under 6 years of age. On the basis of the existing data in
adults and adolescent, in the DEEP-1 trial a pharmacokinetic bridging model was developed to
support the dose selection in children aged less than 6 years affected by transfusion
dependent haemoglobinopathies. The study consisted of two phases, namely an experimental
phase, during which patients received a single dose and a modeling phase, during which PK
data obtained after single dose in patients < 6 years of age were analysed in conjunction
with historical PK data in adults and older children and adolescents. The model-based
analysis of the data obtained after single dose enabled the assessment of the dosing regimen
required for the purpose of accurate pharmacokinetic bridging. The ratio between the
predicted systemic exposure parameters (AUC and Cmax) in the target population and reference
group were used as basis for recommendation of the dose in the target population.
Phase:
Phase 2
Details
Lead Sponsor:
Consorzio per Valutazioni Biologiche e Farmacologiche