The study intends to provide a prospective validation and clinical evaluation of safety and efficacy of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.
Prospective multicentre randomised, placebo-controlled double-blind trial to verify the effects of the administration of probiotics on intestinal microflora in paediatric patients with Cystic Fibrosis.
A double blind, randomised, multicentre, active controlled, parallel-group, phase III trial to evaluate the efficacy, safety and pharmacokinetics of intravenous clonidine (hydrochloride) compared to midazolam for sedation in children from birth to less than 18 years of age. The study is aimed at comparing a new paediatric formulation of clonidine with midazolam. It is ongoing and involves 5 EU countries.
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. The study was concluded on February 2014, providing scientific evidence that the dosage of deferiprone used in adults can provide sufficient exposure to ensure efficacy also in small children and allowing children aged under 6 years of age to start to be recruited in DEEP-2 Safety/Efficacy Study. The study has enrolled a total of 23 patients, of which 18 were considered as evaluable.
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate the efficacy and safety of deferiprone compared to deferasirox in paediatric patients aged from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies. The study is aimed at comparing for the first time the efficacy and safety of deferiprone (DFP) vs deferasirox (DFX) in paediatric patients affected by hereditary haemoglobinopaties requiring chronic transfusions and chelation. The trial is ongoing and involves 344 paediatric patients starting from 1 month of age in EU and non-EU recruiting sites.