DEEP‐2 2012.

Trial name or title	Efficacy and safety study to compare deferiprone versus deferasirox in paediatric patients
Methods	Multicentre, randomised, open label, non‐inferiority active‐controlled study
Participants	Estimated enrolment: 344 Inclusion criteria; People of both genders aged from 1 month up to less than 18 years at the time of enrolment People affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassaemia syndromes and sickle cell disease People on current treatment with DFO or DFX or DFP in a chronic transfusion program receiving at least 150 mL/kg/year of packed red blood cells (corresponding approximately to 12 transfusions) For participants naive to chelation treatment: participants that have received at least 150 mL/kg of packed red blood cells (corresponding to approximately 12 transfusions) in a chronic transfusion program and with serum ferritin levels ≥ 800 ng/mL For participants aged from 1 month to less than 6 years: known intolerance or contraindication to DFO; Written informed consent and participant's informed assent, relating to his/her comprehension abilities and level of maturity Exclusion criteria: Participants with known intolerance or contraindication to either DFP or DFX Participants receiving DFX at a dose > 40 mg/kg/day or DFP at a dose > 100 mg/kg/day at screening Platelet count < 100.000/mm³ during the run‐in phase Absolute neutrophils count < 1.500/mm³ during the run‐in phase Hb levels lower than 8 g/dL during the run‐in phase Evidence of abnormal liver function Iron overload from causes other than transfusional haemosiderosis Severe heart dysfunction secondary to iron overload Serum creatinine level > ULN for age during the run‐in phase History of significant medical or psychiatric disorder The patient has received another investigational drug within 30 days prior to this study Fever and other signs/symptoms of infection in the 10 days before baseline assessment Concomitant use of trivalent cation‐dependent medicinal products such as aluminium‐based antacids Positive test for β‐HCG
Interventions	Experimental: DFP oral solution Comparator: DFX
Outcomes	Primary outcome measure Percentage of successfully chelated participants assessed by serum ferritin levels (all participants) and cardiac MRI T2* (participants above 10 years of age able to have an MRI scan without sedation) Secondary outcome measures LlC as measured by MRI in participants able to undergo MRI scan without sedation Safety and tolerability assessments QoL
Starting date	Date of first enrolment: 29/11/2012
Contact information	Direzione Scientifica via Luigi Porta, 14 27100 Pavia Italy Arianna Gambino, M.Sc. phone: +39.0382.25075 email: deep.2@deep‐project.net / agambino@cvbf.net
Notes	Estimated study completion date: December 2014 Estimated primary completion date: December 2014 (Final data collection date for primary outcome measure)