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 of age affected by transfusion-dependent haemoglobinopathies

CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE0 个研究点目标入组 435 人开始时间: 2012年10月11日最近更新: 2021年12月21日

试验速览

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•1. Patients 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); 2. For patients naïve to chelation treatment: patients 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; 3. For patients aged from 1 month to less than 6 years: known intolerance or contraindication to deferoxamine; 4. Written informed consent and patient's informed assent to patient’s maturity and understanding
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 344
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Patients with known intolerance or contraindication to either DFP or DFX 2. Patients receiving DFX at a dose > 40 mg/kg/day or DFP at a dose > 100 mg/kg/day at screening 3. Platelet count <100.000/mm3 during the run-in phase 4. Absolute neutrophils count <1.500/mm3 during the run-in phase 5. Hb levels lower than 8g/dL during the run-in phase 6. Evidence of abnormal liver function 7. Iron overload from causes other than trasfusional haemosiderosis 8. Severe heart dysfunction secondary to iron overload 9. Serum creatinine level > ULN for age during the run-in phase 10. History of significant medical or psychiatric disorder 11. The patient has received another investigational drug within 30 days prior to this study 12. Fever and other signs/symptoms of infection in the 10 days before baseline assessment 13. Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids 14. Positive test for ß-HCG

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