Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients

• Status: Completed
• Trial end date: 2017-09-21
• Target enrollment: 0
• Participant gender: All

Summary

Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40 mg/kg/day in paediatric patients (1 month < 18 years old) affected by hereditary haemoglobinopathies and requiring frequent transfusions and chelation.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Consorzio per Valutazioni Biologiche e Farmacologiche
• Collaborator: European Commission
• Treatments: Deferasirox; Deferiprone

Criteria

Inclusion Criteria:

• Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment

• Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease

• Patients on current treatment with deferoxamine (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 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;

• Until availability of results from the PK Study (Study DEEP-1, EudraCT n. 2012-000658-67) for patients aged from 1 month to less than 6 years: known intolerance or contraindication to DFO;

• Written informed consent and patient's informed assent, relating to his/her comprehension abilities and level of maturity

Exclusion Criteria:

• Patients with intolerance or known contraindication to either DFP or DFX

• Patients 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 8g/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 (Upper Limit of Normal) 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 clinical trial

• 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 (Human chorionic gonadotropin) and lactating female patients