DEEP – DEFERIPRONE EVALUATION IN PAEDIATRICS FP7-HEALTH- F4-2010-261483

Title: DEEP – DEFERIPRONE EVALUATION IN PAEDIATRICS

Grant agreement: 261483, FP7-HEALTH- F4-2010

Funded by: European Commission (FP7)

Project website: www.deepproject.eu

Duration: 1h 30 m (15.00 - 16.30)

Date: 2nd October, 2014

 

Recipients: project managers, researchers, health institutions and professionals

 

 

Name of the organisation and role in the project: 

Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari – PB (Italy) – Partner

 

Brief project presentation:

The main goal of the DEEP project is the development of the paediatric use of deferiprone for the treatment of iron overload in paediatric patients affected by congenital, transfusion-dependent anaemias such as β-thalassaemia major, also known as “Mediterranean anaemia”, and sickle cell disease (SCD).

Congenital haemoglobinopathies are the most common congenital rare diseases in the world. Mediterranean population are specifically affected with a variable trend depending by immigration movements and fluxes. Patients survival and quality of life depends by regular red blood cells transfusions and by the use of iron chelators to reduce the iron overload.

Deferiprone is the first oral iron chelator approved in Europe and in other Mediterranean countries. After this drug entered the market many advantages have been reported in patients affected by thalassemia and other haemoglobinopathies because of better compliance and increased effect at cardiac level, dealing to lowered mortality.

The DEEP project has been designed specifically for children, starting from the youngest and including adolescents. It is aimed to better define the safety, the tolerability and the efficacy of this drug in order to make its anticipated therapeutic benefits available to the entire paediatric population. Thus, the primary objectives of the DEEP Project are:

a) To perform pharmacokinetics, efficacy and safety studies of deferiprone in children in order to provide data to be included in an updated Summary of Product Characteristics. (DEEP1 and DEEP2)

b) To provide a new formulation specifically tested for young children. The new formulation is of a different strength (80 mg/ml, oral solution) from what is currently marketed (Ferriprox 100 mg/ml, oral soluzione) and has improved flavour in order to increase acceptance (and consequently compliance) by young children. (DEEP 2)

c) To provide longterm safety data on all the current uses (including combination with Deferoxamine) in a large perspective/retrospective population. (DEEP 3)

The decision of the European Commission to fund this plan of research is compliant with the recent European Regulation on Paediatric Medicines that requests that all medicines with a potential to benefit children should be appropriately studied in this population. On the 11th November, 2011 the clinical development described in the project received a positive opinion by the Paediatric Committee (PDCO) at the European Medicines Agency (EMA).

Noticeably the project involves 17 Partners from 6 EU countries and 4 non-EU, all representing the main geographic area affected by congenital haemoglobinopathies. Apulia is strongly involved in this disease and the Policlinic of Bari is performing a key-role in terms of management and clinical activities, in particular acting as the Coordinating Centre of the DEEP-1 Clinical Trial and hosting the Drug Management Service for all the Italian recruiting centres.

 

Partners:

Consorzio per Valutazioni Biologiche e Farmacologiche - CVBF (Italy) – Coordinator

Leiden University – UL (The Netherlands)

Azienda Ospedaliera di Rilievo Nazionale “Antonio Cardarelli” – AOC (Italy)

National and Kapodistrian University of Athens – NKUA (Greece)

University Hospital Center “Mother Teresa” – UHCT (Albania)

Medical and Public Health Service – MPHS (Cyprus)

Cairo University – CU (Egypt)

ApoPharma Inc. (Canada)

Fondazione Italiana “Leonardo Giambrone” per la Guarigione della Talassemia (Italy)

Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello – AORC (Italy)

Azienda Ospedaliera di Padova – AODP (Italy)

Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari – PB (Italy)

Apotex Inc. (Canada)

University Hospital Erlangen – UKER (Germany)

Fondazione per la Ricerca Farmacologica “Gianni Benzi” Onlus – FGB (Italy)

Centre National de Greffe de Moelle Osseuse Tunis – CNGMO (Tunisia)

University College of London – UCL / Barts and the London NHS Trust (UK)

 

Agenda

 

15:00 Chairs: P. Giordano – C. De Lorenzo

  • Best practices in paediatric clinical research: the DEEP experience (D. Bonifazi)
  • Participating in an international research network: the experience of the Policlinic of Bari (V. Dattoli)
  • Results from the PK/PD clinical trial and patients’ recruitment for the efficacy/safety and long-term safety studies (G. Del Vecchio)
  • Responsibilities of the Hospital Pharmacy of the ‘Azienda Ospedaliero-Universitaria Policlinico’ of Bari: innovative roles and tasks (M. Lattarulo)

16:00 Discussion

  • Ludovico Abbaticchio – Member of the Board of Administration in AIFA (‘Agenzia Italiana del Farmaco’)
  • Adriana Ceci – DEEP Project Scientific Coordinator
    • Gaetano Lodeserto – Vice president of ’Associazione per la Guarigione della Thalassemia Manduria (TA)
    • Giuseppe Mele – Member of the Board of Directors of UNITED (Federazione Nazionale dei Thalassemici e Drepanocitici)

Resumes

 Dr. Ludovico Abbaticchio

Ludovico Abbaticchio was born in 1953, he graduated in Medicine and Surgery at the ' Università degli Studi di Bari’ and specialized in obstetrics and gynecology, with particular focus on individual, couple and famlily’s health. He is an expert in Policies of the juvenile health and Urgent Care physician (1980-1984.  He practiced his expertise as Gynecologist at AIED , the Italian Association for the Demographic Education (1983-1987). He has been working as general practicioner since 1984.  He was Health Inspector In 1987 and founder of AIECS, ‘Associazione italiana educazione contraccettiva sessuale’, and president from 1988 and 1993. He is the author of several scientific publications . He was elected as member of the City Council of Bari in 1990 and the Commissions for Culture and Social Services. He embodied the Director of the Medical Association of Bari (1997-1999). He is at the summit of the Labor Union C.U.M.I. (Confederazione Unitaria Medici Italiani). He is Group Leader DS in the City Council, Vice president of the Urban Commission and Regional President of the labor union for Italian physicians. He is member of the Bioethics Commission ASL Bari. On August 28th 2014, he was presented to the Italian Council of Ministers of the as Member of the Board of Directors of AIFA (Agenzia Italiana del Farmaco).

Dr. Donato Bonifazi

Donato Bonifazi is the Managing Director of the Consorzio per Valutazioni Biologiche e Farmacologiche -CVBF-  (composed by the ‘Università degli Studi di Bari Aldo Moro’, the Gianni Benzi Pharmacological Research Foundation, the Italian Group for Pharmacoeconomic Studies, Bioikos Farma Srl and Pharmaceutical Research Management Srl) the Corporate Secretary of the Gianni Benzi Pharmacological Research Foundation and member of EFGCP, AICRO and EUCROF.

He is an economist specialised in European Project Management and in Clinical Trials Management, currently engaged as:

- Project Manager of the FP7 Project DEEP (DEferiprone Evaluation in Paediatrics), involving European, Canadian and North-African Partners;

- WP Leader for the trial management in the FP7 Project GAPP (GApapentin in Paediatric Pain);

- collaborator in the FP7 Projects CloSed, GRiP, NeoVanc and Neomero.

He is also an expert in networking and communication activities, in charge of the dissemination activities for the InNerMeD-I-Network (DG SANCO Project) on behalf of the Gianni Benzi Foundation and responsible for the long-term development of the Clinical-Biological Integrated Regional Network for Rigenerative Medicine - RIMedRI (Sicily Region Project) on behalf of the Italian National Centre for Biological Resources.

 

Prof. Adriana Ceci

Paediatrician, Pharmacologist, and Professor at the University of Bari until November 2013, Adriana Ceci is the Coordinator of the CVBF Scientific Committee, President of the Gianni Benzi Pharmacological Research Foundation (since 2007), and a member of the European Medicines Agency’s Paediatric Committee (since 2008).
Professor Ceci is the CVBF Scientific Coordinator in GAPP (GAbapentin in Paediatric Pain) FP7 Project, member of the Editorial Board and the Executive Board of the FP7 GRIP (Global Research in Paediatrics) Network of Excellence and Coordinator of the TEDDY (Task-Force in Europe for Drug Development for the Young) Network of Excellence, which has been created as an FP6 Project, still functioning as a cooperative network. She is the author of numerous scientific papers in peer reviewed journals and a referee for national and international journals.

   Dr. Vitangelo Dattoli

Vitangelo Dattoli embodies the General Director of the ‘Azienda Ospedaliero-Universitaria Consorziale Policlinico’ of Bari since 2006.

He graduated in Medicine and Surgery at the ‘Università degli Studi di  Bari Aldo Moro’ in 1984 with a final grade of 110/110 and in December 1984 he achieved qualification as Physician. In 1987 he received the Diploma of Specialist in Forensic Medicine and Insurance with 50/50 cum laude and in 1991 he reached the Diploma of Specialist in Hygiene and hospital Techniques with 50/50 and honors at the ‘Università degli Studi di Bari Aldo Moro’ where he has been carrying out its teaching activity since 2001 (Scuola di specializzazione in Igiene, Scuola di specializzazione in Criminologia Clinica e Psicologia Forense, Scuola di Specializzazione in Medicina Legale e delle Assicurazioni, Master in Criminologia Perfezionamento in Diritto Sanitario) and at the ‘Università degli Studi di Foggia’ (Scuola di Specializzazione in Medicina Legale e delle Assicurazioni, Master in Management Infermieristico).  

He is currently member of the Council for Economic Affairs of the Ente Ecclesiastico – Ospedale Generale Regionale "F. Miulli" located in  Acquaviva delle Fonti (Bari - Italy).

Dr. Ciro De Lorenzo

Ciro De Lorenzo is the President of the Associazione Talassemici Taranto, Italy.

 

Dr. Giovanni Carlo Del Vecchio

Giovanni Carlo Del Vecchio is Doctor at the U.O. Pediatria “F. Vecchio” of the ‘Azienda Ospedaliero‐Universitaria Consorziale Policlinico’ of Bari, where he graduated in Medicine and Surgery with a final grade of 110/10 cum laude, in 1988. He achieved the Diploma of Specialist in Paediatrics in 1992, and the Diploma of Specialist in Haematology in 2004 with highest hounour and laude. He won a scholarship and attended the ‘Laboratory of Experimental Hematology and Oncology – University of Düsseldorf’ (Germany) from November 1992 to May 1993. At the U.O. Pediatria “F. Vecchio” of the ‘Azienda Ospedaliero‐Universitaria Consorziale Policlinico’ of Bari  is in charge of conducting clinical activities at the Department of paediatric haematology-oncology, the Day Hospital of paediatric haematology, the Regional Center for Rare Anemia ’Anemie Ereditarie nel Bambino’. He is Professor of paediatrics in the courses of Bachelor's Degree, Supplementary teaching for students of Medicine and post-graduate students of Paediatrics at the ‘Università degli Studi di Bari Aldo Moro’. He has been carrying out research activities, throughout the conduction of national and international GCP-compliant-studie sin the field of Paediatrics, with particular reference to paediatric haematology as documented in over 60 publications on national and international journals. He is a member of the strategic committee ‘Patologia del Globulo Rosso’ and ‘Difeti della Coagulazione’ at AIEOP. He actively contribuited to the writing of the National Guidelines for SCD and ITP in paediatrics and for diagnosis and treatment fo the thrombotic complications during acute lymphoblastic leukemia in paediatrics.

Prof.  Paola Giordano

Paola Giordano graduated in Medicine and Surgery at the ‘Università degli Studi di Bari Aldo Moro’ in 1986 in the first session of the sixth year of the study course with a final grade of 110/110 cum laude. In November 1986 she has achieved the professional qualification with 110/110. She attended the School of Specialisation in Paediatrics and achieved the Diploma of Specialist on 27/10/1990 with a grade of 50/50 cum laude. In January 1990 she won one of the biennial scholarship allocated by the CNR for the south of Italy, the National Committee for biological and medical sciences at the Research Center of Experimental Endocrinology and Oncology of the Second Faculty of Medicine of Naples, directed by Prof. Gaetano Salvatore. As a CNR fellow she attended for six months from February to July 1990, the laboratory of Haematology at the Hospital ‘Necker - Enfants Malades’ in Paris. On 01/04/1992 she was the winner of the national competition for a position of the research group FO4, focused on General Pathology (FO4A) and Medical Oncology (FO4C). She opted for the Department of Biomedicine of Developmental Age. She is the author of more than 200 papers concerning the paediatric haemo-oncology with particular reference to the mechanisms of haemostasis. She has been carrying out the teaching activities since 1990 at the ‘Università degli Studi di Bari Aldo, Faculty of Medicine (Bachelor in Medicine and Surgery, Bachelor of Science in Orthoptics and assistant in ophthalmology, Bachelor of Science in Biomedical Laboratory Technician ,  degree in professional educators) in the School of Specialisation in Paediatrics. She is the Director of the School of Specialisation in Paediatrics at the ‘Università degli Studi di Bari Aldo Moro’.

 

Dr. Michele Lattarulo

Michele Lattarulo embodies the Director of “Raggruppamento Unità Operative di Farmacia” at the Azienda Ospedaliero-Universitaria “Consorziale” Policlinic of Bari since 1993, he graduated in Pharmacy at the University of Bari in 1975. 

In December 1977 he received the Diploma of Specialist in Pharmacognosy at the ‘Università degli Studi di Messina’ and in December 1984 he achieved the Diploma of Specialist in Hospital Pharmacy at the University of Bari.

He is currently:

-Professor of "Clinical Pharmacy" at the University of Bari (School of Specialisation in Hospital Pharmacy) and responsible for the coordination and mentoring of the qualifying activities of the same school since 1994;

- Professor of Nutritional Sciences at the University of Bari (Postgraduate School of Gastroenterology - Faculty of Medicine) since 1998;

- member of the Scientific Committee, Professor and tutor of the Postgraduate Degree in "Pharmacist of Department" - at the University of Bari since 2010;

- Professor of "Managerial activities in Artificial Nutrition" at the University Master's Degree in "Manager of Pharmaceutical Department" at the ‘Università degli Studi di Camerino’ since 2001;

He is author of 3 monographs on artificial Nutrition and has collaborated at the drafting of more than 140 national and international publications.

Dr. Gaetano Lodeserto

 

Gaetano Lodeserto is Vice president of ’Associazione per la Guarigione della Thalassemia Manduria (TA).

 Dr. Giuseppe Mele

Giuseppe Mele is member of the Board of Directors of UNITED (‘Federazione Nazionale dei Thalassemici e Drepanocitici’)

 

 

 

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