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DEferiprone Evaluation in Paediatrics

Objective

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.

Field of science

  • /medical and health sciences/clinical medicine/paediatrics
  • /natural sciences/chemical sciences/inorganic chemistry/inorganic compounds

Call for proposal

FP7-HEALTH-2010-single-stage
See other projects for this call

Funding Scheme

CP-FP - Small or medium-scale focused research project

Coordinator

CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE
Address
Via Nicolo Putignani 178
70122 Bari
Italy
Activity type
Research Organisations
EU contribution
€ 1 569 532,79
Administrative Contact
Donato Bonifazi (Dr.)

Participants (17)

THE SCHOOL OF PHARMACY, UNIVERSITY OF LONDON

Participation ended

United Kingdom
EU contribution
€ 1 635,14
Address
Brunswick Square 29-39
WC1N 1AX London
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ian Chi Kei Wong (Prof.)
UNIVERSITEIT LEIDEN
Netherlands
EU contribution
€ 245 628,35
Address
Rapenburg 70
2311 EZ Leiden
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ton Brouwer (Dr.)
HOPITAL D'ENFANTS DE TUNIS

Participation ended

Tunisia
Address
Place Bab Saadoun
1006 Tunis
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Ridha Ben Othman (Mr.)
AZIENDA OSPEDALIERA ANTONIO CARDARE
Italy
EU contribution
€ 187 970,55
Address
Via Antonio Cardarelli 9
80131 Napoli
Activity type
Research Organisations
Administrative Contact
Elia Abbondante (Dr.)
ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON
Greece
EU contribution
€ 267 099,77
Address
6 Christou Lada Str
10561 Athina
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Efi Kafentzi (Ms.)
QENDRES SPITALORE UNIVERSITARE NENETEREZA TIRANE
Albania
EU contribution
€ 162 429,50
Address
Rr Dibres 370
1001 Tirana
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Ariel Como (Dr.)
Ministry of Health of the Republic of Cyprus
Cyprus
EU contribution
€ 197 394,74
Address
1 Prodromou Street & 17 Chilonos Street 1 & 17
1448 Nicosia
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Missouri Skevoula (Mrs.)
CAIRO UNIVERSITY
Egypt
EU contribution
€ 681 177,99
Address
Al Orman Guiza
12613 Giza
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Amal El-Beshlawy (Prof.)
APO-PHARMA INC CORP
Canada
EU contribution
€ 20 500
Address
Signet Drive 150
M9L 1T9 Toronto
Activity type
Other
Administrative Contact
Michael Spino (Dr.)
FONDAZIONE ITALIANA LEONARDO GIAMBRONE PER LA GUARIGIONE DELLA THALASSEMIA
Italy
EU contribution
€ 95 284,40
Address
Via Girolamo Savonarola 19
00195 Roma
Activity type
Research Organisations
Administrative Contact
Angela Iacono (Dr.)
AZIENDA OSPEDALIERA OSPEDALI RIUNITI VILLA SOFIA-CERVELLO
Italy
EU contribution
€ 388 582,94
Address
Via Strasburgo 233
90146 Palermo
Activity type
Research Organisations
Administrative Contact
Daniela Faraoni (Dr.)
AZIENDA OSPEDALE UNIVERSITA PADOVA
Italy
EU contribution
€ 323 956,23
Address
Via Nicolo Giustiniani 1
35128 Padova
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Administrative Contact
Maria Caterina Putti (Dr.)
AZIENDA OSPEDALIERO UNIVERSITARIA CONSORZIALE POLICLINICO DI BARI
Italy
EU contribution
€ 289 646,05
Address
Piazza Giulio Cesare 11
70124 Bari
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Pasquale Cassese (Dr.)
APOTEX INC CORPORATION
Canada
Address
Signet Drive 150
M9L 1T9 Toronto Ontario
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Administrative Contact
Sheron Khan (Dr.)
FONDAZIONE PER LA RICERCA FARMACOLOGICA GIANNI BENZI ONLUS
Italy
EU contribution
€ 293 985,99
Address
Via Abate Eustasio 30
70010 Valenzano Bari
Activity type
Research Organisations
Administrative Contact
Rosa Conte (Dr.)
UNIVERSITATSKLINIKUM ERLANGEN
Germany
EU contribution
€ 261 512,80
Address
Maximiliansplatz 2
91054 Erlangen
Activity type
Higher or Secondary Education Establishments
Administrative Contact
Katrin Faber (Dr.)
CENTRE NATIONAL DE GREFFE DE MOELLE OSSEUSE
Tunisia
EU contribution
€ 276 625,76
Address
Rue Djebel Lakhdar 02 - Bab Saadoun
1006 Tunis
Activity type
Research Organisations
Administrative Contact
Mohamed Lassaad Ayadi (Mr.)