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 - DEEP-2

Consorzio per Valutazioni Biologiche e Farmacologiche0 个研究点目标入组 344 人开始时间: 2013年4月10日最近更新: 2015年10月12日

适应症

试验速览

暂无简介。

•1. Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment;
•2. Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease;
•3. 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);
•4. 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;
•5. For patients aged from 1 month to less than 6 years: known intolerance or contraindication to deferoxamine;
•6. Written informed consent and patient's informed assent according 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
•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 beta-HCG or lacting female patients.

发起方