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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.
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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)

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THE SCHOOL OF PHARMACY, UNIVERSITY OF LONDON

United Kingdom

EU Contribution

€ 1 635,14

UNIVERSITEIT LEIDEN

Netherlands

EU Contribution

€ 245 628,35

HOPITAL D'ENFANTS DE TUNIS

Tunisia

AZIENDA OSPEDALIERA ANTONIO CARDARE

Italy

EU Contribution

€ 187 970,55

ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON

Greece

EU Contribution

€ 267 099,77

QENDRES SPITALORE UNIVERSITARE NENETEREZA TIRANE

Albania

EU Contribution

€ 162 429,50

Ministry of Health of the Republic of Cyprus

Cyprus

EU Contribution

€ 197 394,74

CAIRO UNIVERSITY

Egypt

EU Contribution

€ 681 177,99

APO-PHARMA INC CORP

Canada

EU Contribution

€ 20 500

FONDAZIONE ITALIANA LEONARDO GIAMBRONE PER LA GUARIGIONE DELLA THALASSEMIA

Italy

EU Contribution

€ 95 284,40

AZIENDA OSPEDALIERA OSPEDALI RIUNITI VILLA SOFIA-CERVELLO

Italy

EU Contribution

€ 388 582,94

AZIENDA OSPEDALIERA DI PADOVA

Italy

EU Contribution

€ 323 956,23

AZIENDA OSPEDALIERO UNIVERSITARIA CONSORZIALE POLICLINICO DI BARI

Italy

EU Contribution

€ 289 646,05

APOTEX INC CORPORATION

Canada

FONDAZIONE PER LA RICERCA FARMACOLOGICA GIANNI BENZI ONLUS

Italy

EU Contribution

€ 293 985,99

UNIVERSITATSKLINIKUM ERLANGEN

Germany

EU Contribution

€ 261 512,80

CENTRE NATIONAL DE GREFFE DE MOELLE OSSEUSE

Tunisia

EU Contribution

€ 276 625,76

Project information

Grant agreement ID: 261483

Status

Closed project

  • Start date

    1 January 2011

  • End date

    31 August 2016

Funded under:

FP7-HEALTH

  • Overall budget:

    € 7 626 002,90

  • EU contribution

    € 5 262 963

Coordinated by:

CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE

Italy

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