Obiettivo
β-thalassaemia major is one of the most severe forms of chronic congenital anaemia. The recommended treatment consists in regular blood transfusions combined with chelating therapy to remove harmful iron accumulation in the body. The use of deferoxamine, the first chelating agent only available for subcutaneous administration is limited due to toxicity and the lack of compliance, despite its satisfactory therapeutic effects. An oral iron chelating agent, deferiprone, was authorised in Europe in August 1999 and recommended for the treatment of iron overload in patients with thalassaemia major when deferoxamine is contraindicated or inadequate. Despite a wide experience of the administration of deferiprone for thalassaemic patients, limited data are available on its use in children below 10 years and the need for additional data in this age subset was clearly indicated in the 2009 priority list approved by the Paediatric Committee at the European Medicines Agency (PDCO). In addition, according to the recent scientific advancements and in consideration of the anticipated benefit of this chelator in controlling cardiac iron overload, studies evaluating the effects of the deferiprone in all the paediatric ages and in all transfusion-dependent chronic congenital anaemia (including Sickle Cell Diseases) were also considered a critical therapeutic need.
The DEEP project, in line with these premises, has been funded with the specific aim to produce a new oral liquid formulation of deferiprone suitable for the paediatric use and to provide evidences for the use of this chelator as first line therapy in the whole paediatric population (from 1 month to 18 years) affected by transfusion-dependent chronic anaemia.
The condition under study in the DEEP project is rare. This poses special difficulties in the conduct of the studies due to the small patient population and the need to involve a large number of recruiting centres . However, being dedicated to develop an orphan drug, DEEP has been also recognised in the context of IRDiRC, the International Rare Diseases Research Consortium devoted to repurpose/develop 200 new drugs for Rare Diseases by the end of 2020.
Main features of the DEEP project are:
-The innovative design of the clinical studies including pharmacokinetic modelling for the definition of the most appropriate dosage of deferiprone in younger children, the cardiac MRI T2* evaluation as primary endpoint, a three years safety study aimed at evaluating deferiprone, in monotherapy or in combination, in the real world's setting and, for the first time, a comparative efficacy-safety trial to compare the two existing oral chelators: deferiprone and deferasirox.
-The DEEP Consortium including European and non-European Countries from the Mediterranean region where the transfusion-dependent congenital anaemia, in particular β-thalassemia major, is particularly widespread: the collaboration within a multinational and multicultural network makes the Project extremely challenging due to many different ethical, methodological and social approaches to be explored and positively addressed.
Campo scientifico
Invito a presentare proposte
FP7-HEALTH-2010-single-stage
Vedi altri progetti per questo bando
Meccanismo di finanziamento
CP-FP - Small or medium-scale focused research projectCoordinatore
70122 Bari
Italia
Mostra sulla mappa
Partecipanti (17)
Partecipazione conclusa
WC1N 1AX LONDON
Mostra sulla mappa
2311 EZ Leiden
Mostra sulla mappa
Partecipazione conclusa
1006 Tunis
Mostra sulla mappa
80131 Napoli
Mostra sulla mappa
10561 Athina
Mostra sulla mappa
1001 Tirana
Mostra sulla mappa
1448 Nicosia
Mostra sulla mappa
12613 Giza
Mostra sulla mappa
M9L 1T9 Toronto
Mostra sulla mappa
00195 ROMA
Mostra sulla mappa
90146 PALERMO
Mostra sulla mappa
35128 Padova
Mostra sulla mappa
70124 Bari
Mostra sulla mappa
M9L 1T9 TORONTO ONTARIO
Mostra sulla mappa
70010 Valenzano Bari
Mostra sulla mappa
91054 Erlangen
Mostra sulla mappa
1006 TUNIS
Mostra sulla mappa