Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Condition: Chronic Iron Overload
MeSH terms: Hemoglobinopathies; Iron Overload
Study type: Interventional
Phase: Phase 3

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.
    

Detailed description: 
      Haemoglobinopathies are a group of inherited disorders characterized by structural variations
      of the haemoglobin molecule. Most of the patients affected require for survival chronic red
      blood cells transfusions to overcome ineffective erythropoiesis. Unfortunately, all
      chronically transfused patients become clinically iron overloaded as there is no
      physiological mechanism for the removal of iron from the body. The pathologic changes and
      clinical manifestations associated to chronic iron overload are common among all
      transfusional iron-overload patients, albeit best documented in patients with
      beta-thalassemia major. The recommended treatment consists in regular blood transfusions
      combined with chelating therapy to remove the harmful iron accumulation in the body.

      Currently, in the clinical practice particularly in children and adolescents, the criteria
      leading to the choice of the chelating agent include also the adherence to therapy, thus
      favouring the use of oral chelators (Ceci A et al., 2011) DFP (Deferiprone) was the first
      oral chelator authorised in Europe in 1999 as second line treatment for the treatment of iron
      overload in patients with thalassaemia major when DFO (Deferoxamine) is contraindicated or
      inadequate. However, despite a wide experience of DFP with iron overloaded (specifically
      thalassaemic )patients, limited data are available for younger children. For this reason the
      need for additional data in younger children is expressively included in the 2009 PDCO
      (Paediatric Committee) Priority List.

      The purpose of this study is to assess the non-inferiority of DFP compared to DFX
      (deferasirox)in paediatric patients affected by hereditary haemoglobinopathies requiring
      chronic transfusions and chelation. Non inferiority will be established in terms of
      percentage of patients successfully chelated, as assessed by serum ferritin levels (in all
      patients) and cardiac MRI T2* (in patients above 10 years of age able to have an MRI scan
      without sedation).
    

Intervention name(s): Deferiprone; Deferasirox
Intervention type(s): Drug; Drug
Intervention description(s): Deferiprone 80 mg/mL oral solution; Deferasirox is used at the following dosage strengths: 125 mg, 250 mg and 500 mg
Other intervention name(s): DFP; DFX; ATC Code:V03AC03
Intervention MeSH terms: Deferasirox; Deferiprone
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: chronic iron overload; hereditary haemoglobinopathy; beta thalassaemia major; chelating agents; deferiprone; deferasirox; children; paediatrics