Chronic Iron Overload Clinical Trial
Official title:
Multi-centre, Oral Single Dose Experimental and Modelling Study to Evaluate the Pharmacokinetics of Deferiprone in Patients Aged From 1 Month to Less Than 6 Years of Age Affected by Transfusion-dependent Haemoglobinopathies.
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 will be developed to support the dose selection in children aged less than 6 years. The study will consist of two phases, namely an experimental phase, during which patients will receive a single dose and a modeling phase, during which PK data obtained after single dose in patients < 6 years of age will be 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 will enable 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 will be used as basis for recommendation of the dose in the target population.
n/a
Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label
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