Less than half of all authorised medicines commonly used in children have been properly tested in this group, and running clinical trials involving children is hard.
The conect4children (c4c) project aims to create a sustainable, integrated pan-European collaborative paediatric network that will speed up and facilitate the running of high quality clinical trials in children while ensuring that the voices of young patients and their families are heard. It will build the capacity for conducting multinational paediatric clinical trials for all disease areas and all phases of the clinical drug development process.
One focus of the project is to promote innovative trial designs that will support drug development for rare paediatric diseases and areas of high medical need. The project will also set up an education and training platform for future leaders in paediatric drug development.
Ultimately, the paediatric clinical trials network will contribute to the development of better medicines for babies, children and young people.
Achievements & News
IMI project c4c has pioneered an advice process involving diverse experts including patients to aid drug developers when designing paediatric...
c4c aims to enable hospitals and clinical sites across Europe to become ‘trial ready’ for paediatric clinical trials. We asked...
c4c aims to enable hospitals and clinical sites across Europe to become ‘trial ready’ for paediatric clinical trials. The project has just embarked on a number of proof of viability (PoV) studies, where they use clinical trials to build and test the network.###
‘The studies are testing the ability of the c4c network to provide information and advice that is not always available to sponsors (the organisation responsible for overseeing and/or funding the study),’ explained the project leadership team in an interview with the IMI Programme Office.
‘Investigators (the person/people responsible for carrying out the study at a site) sometimes find interaction with sponsors and contract research organisations (the organisation contracted by the sponsor to perform some of their duties) difficult. By allowing clinicians to talk with clinicians, the c4c network facilitates site identification, feasibility, and setup, including troubleshooting delayed ethics applications, site understanding of budgets and contracts, and trial procedures at site level.'
The project has also set up advice groups in as well as a public involvement database comprising individual parents / patients and patient organisations.
‘The c4c network promotes the central role of participants in paediatric trials by supporting children, young people, and their families in activities such as the review of study protocols, to ensure their opinions and needs are incorporated in the design and implementation of studies,’ the project said.
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Three IMI projects – c4c, EUPATI and PREFER – are cited in a new Orphan Drug Development Guidebook released by the International Rare Diseases Research Consortium (IRDiRC). ###Developing ‘orphan drugs’ (i.e. drugs for rare diseases) is highly challenging, and just 5 % of rare diseases have an approved treatment. The IRDiRC describes the new guidebook as ‘a patient focused guidebook that describes the available tools, incentives, resources and practices for developing traditional and innovative drugs/therapies for rare diseases and how to best use them.’ It lists a range of resources from organisations around the world, including IMI.
c4c aims to generate a sustainable infrastructure that optimises the delivery of clinical trials in children, and the guidebook suggests this could be useful for those developing medicines for rare paediatric diseases.
On EUPATI, the guidebook describes its patient education resources as ‘very relevant to rare disease medicines development and highly useful and appreciated by the rare disease patients that finished the academy and use the toolbox’.
On PREFER, the guide highlights how patient preference studies can be useful during regulatory benefit-risk assessment for certain drugs in several major ways.
The guidebook is presented in an article in Nature Reviews Drug Discovery. ‘By enhancing the use of available tools, delays in development timelines can be avoided, risks and costs reduced, and patient and regulatory acceptability improved,’ the authors conclude.
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IMI’s c4c project is inviting children and young people to help decide how clinical trials with child participants should be run. Children taking part in a clinical trial, depending on their maturity and understanding, have to sign an assent form ###declaring that they understand the purpose of the study and its benefits and risks, and that they agree to participate. However, these forms are not always child friendly. In an interview with the IMI Programme Office, Joana Claverol of the Hospital San Joan de Déu in Barcelona explains how she and her colleagues are setting up a European network of advisory groups made up of children and young people with different diseases to get their help to improve the different research projects developed at the hospital.
‘We want these children to participate not only as participants in the trial, but to act as advisors on trial design, to review protocols, to assess patient documentation, and to talk about paediatric and medical needs,’ she says. The network will contribute to the project’s goal of building a network of hospitals in multiple countries that can undertake high-quality clinical trials for children, across all paediatric age groups and diseases in children.
The c4c project has announced its first studies designed to increase our understanding of certain medicines that are commonly used in babies, children and young people.### Currently half of all medicines used in children have never actually been tested in children; c4c is working to change this by creating a pan-European network for paediatric clinical trials. The project has now selected four studies that will leverage the fledgling network and implement new ways to integrate children’s and young people’s views in clinical trial design. They will also apply novel ways of evaluating medicines. The four studies address a range of disease areas and age groups. One will assess the effectiveness of paracetamol in premature babies with a serious heart defect called patent ductus arteriosus. Another will investigate the use of steroids in children with Kawasaki disease, which mainly affects the under 5’s and is characterised by a high temperature, rash, swollen glands in the neck, dry, cracked lips, and red eyes, fingers or toes. The third study focuses on posaconazole in children and young people with cystic fibrosis and an Aspergillus (fungal) infection. Finally, the project will run a trial on losartan in children and youngsters with osteogenesis imperfecta, a genetic disorder which results in fragile bones. All four studies will be led by academic institutions. Additional studies led by industry partners are also planned.
ParticipantsShow participants on map
- Bayer Aktiengesellschaft, Leverkusen, Germany
- Eli Lilly And Company LTD, Basingstoke, United Kingdom
- F. Hoffmann-La Roche AG, Basel, Switzerland
- Glaxosmithkline Research And Development LTD., Brentford, Middlesex, United Kingdom
- Institut De Recherches Internationales Servier Iris, Suresnes, France
- Janssen Pharmaceutica Nv, Beerse, Belgium
- Novartis Pharma AG, Basel, Switzerland
- Pfizer Limited, Sandwich, Kent , United Kingdom
- Sanofi-Aventis Recherche & Developpement, Chilly Mazarin, France
- UCB Biopharma, Brussels, Belgium
Universities, research organisations, public bodies, non-profit groups
- Aristotelio Panepistimio Thessalonikis, Thessaloniki, Greece
- Arsenal.It - Centro Veneto Ricercae Innovazione Per La Sanita Digitale, Treviso Tv, Italy
- Associação para Investigação e Desenvolvimento da Faculdade de Medicina, Lisbon, Portugal
- Azienda Sanitaria Universitaria Friuli Centrale, Udine, Italy
- Conect4children Stichting, Utrecht, Netherlands
- Ecrin European Clinical Research Infrastructure Network, Paris, France
- European Cystic Fibrosis Society, Karup, Denmark
- Fondazione PENTA - for the treatment and care of children with HIV-ONLUS, Padova, Italy
- Fondazione Per La Ricerca Farmacologica Gianni Benzi Onlus, Valenzano Bari, Italy
- Fundacio Privada Per A La Recerca I La Docencia Sant Joan De Deu, Esplugues de Llobregat, Spain
- Gyermekgyogyaszati Klinikai Vizsgaloi Halozat, Budapest, Hungary
- Helse Bergen Hf, Bergen, Norway
- Hus-Yhtyma, Helsinki, Finland
- Institut National De La Sante Et De La Recherche Medicale, Paris, France
- Instytut Pomnik Centrum Zdrowia Dziecka, Warszawa, Poland
- Istituto Giannina Gaslini, Genova, Italy
- Ospedale Pediatrico Bambino Gesu, Rome, Italy
- Rete Italiana Per Le Sperimentazioni Cliniche In Pediatria, Rome, Italy
- Robert Bosch Gesellschaft Fur Medizinische Forschung Mbh, Stuttgart, Germany
- Servicio Madrileno De Salud, Madrid, Spain
- Servizo Galego De Saude, Santiago de Compostela, Spain
- Siop Europe, Brussels, Belgium
- Stichting Radboud Universitair Medisch Centrum, Nijmegen, Netherlands
- Swiss Clinical Trial Organisation (Scto), Basel, Switzerland
- Tartu Ulikool, Tartu, Estonia
- The University Of Liverpool, Liverpool, United Kingdom
- Universita Degli Studi Di Cagliari, Cagliari, Italy
- Universitatsklinikum Heidelberg, Heidelberg, Germany
- Universiteit Gent, Gent, Belgium
- University College Cork - National University Of Ireland, Cork, Cork, Ireland
- University College London, London, United Kingdom
- University Of Newcastle Upon Tyne, Newcastle upon Tyne, United Kingdom
- Univerzita Karlova, Prague 1, Czech Republic
- Västra Götalandsregionen, Vanersborg, Sweden
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
- Okids GMBH, Wien, Austria
- Eurordis - Rare Diseases Europe, Paris, France
- Azienda Ospedaliera Universitaria Meyer Irccs, Firenze, Italy
- Azienda Socia-Sanitaria Territoriale Degli Spedali Civili Di Brescia, Brescia, Italy
- Consorzio Per Valutazioni Biologiche E Farmacologiche, Pavia, Italy
- Consorzio Per Valutazioni Biologiche E Farmacologiche, Pavia, Italy
- Fondazione Irccs Ca' Granda - Ospedale Maggiore Policlinico, Milan, Italy
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario Clinico San Carlos, Madrid, Spain
- Istituto Di Ricovero E Cura A Carattere Scientifico Burlo Garofolo, Trieste, Italy
- Universita Degli Studi Di Padova, Padova, Italy
- Universita Degli Studi Di Roma Tor Vergata, Rome, Italy
- Vseobecna Fakultni Nemocnice V Praze, Praha 2, Czechia
|Name||EU funding in €|
|Aristotelio Panepistimio Thessalonikis||951 951|
|Arsenal.It - Centro Veneto Ricercae Innovazione Per La Sanita Digitale||212 500|
|Associação para Investigação e Desenvolvimento da Faculdade de Medicina||1 845 554|
|Azienda Sanitaria Universitaria Friuli Centrale||476 113|
|Conect4children Stichting||1 112 500|
|Ecnp Research And Scholarship Stichting (De Ecnp Research And Scholarship Foundation) (left the project)||95 789|
|Ecrin European Clinical Research Infrastructure Network||694 672|
|European Cystic Fibrosis Society||185 196|
|Eurordis - Rare Diseases Europe||653 750|
|Fondazione PENTA - for the treatment and care of children with HIV-ONLUS||9 431 802|
|Fondazione Per La Ricerca Farmacologica Gianni Benzi Onlus||2 283 554|
|Fundacio Privada Per A La Recerca I La Docencia Sant Joan De Deu||1 463 531|
|Gyermekgyogyaszati Klinikai Vizsgaloi Halozat||478 965|
|Helse Bergen Hf||754 293|
|Hus-Yhtyma||2 310 627|
|Institut National De La Sante Et De La Recherche Medicale||6 093 980|
|Instytut Pomnik Centrum Zdrowia Dziecka||698 552|
|Istituto Giannina Gaslini||523 004|
|Karolinska Institutet (left the project)||384 396|
|Okids GMBH||999 480|
|Ospedale Pediatrico Bambino Gesu||3 544 928|
|Rete Italiana Per Le Sperimentazioni Cliniche In Pediatria||1 007 181|
|Robert Bosch Gesellschaft Fur Medizinische Forschung Mbh||2 017 146|
|Servicio Madrileno De Salud||450 876|
|Servizo Galego De Saude||2 273 765|
|Siop Europe||1 130 000|
|Stichting Radboud Universitair Medisch Centrum||4 451 546|
|Swiss Clinical Trial Organisation (Scto)||667 680|
|Tartu Ulikool||1 530 554|
|The University Of Liverpool||5 741 617|
|Universita Degli Studi Di Cagliari||441 222|
|Universitatsklinikum Heidelberg||312 283|
|Universiteit Gent||1 565 015|
|University College Cork - National University Of Ireland, Cork||1 150 670|
|University College London||3 622 179|
|University Of Newcastle Upon Tyne||2 203 993|
|Univerzita Karlova||724 900|
|Västra Götalandsregionen||633 761|
|Name||Funding in €|
|Azienda Ospedaliera Universitaria Meyer Irccs||40 000|
|Azienda Socia-Sanitaria Territoriale Degli Spedali Civili Di Brescia||613|
|Consorzio Per Valutazioni Biologiche E Farmacologiche||82 865|
|Consorzio Per Valutazioni Biologiche E Farmacologiche||544 512|
|Fondazione Irccs Ca' Granda - Ospedale Maggiore Policlinico||40 000|
|Fundacion Para La Investigacion Biomedica Del Hospital Universitario Clinico San Carlos||52 010|
|Istituto Di Ricovero E Cura A Carattere Scientifico Burlo Garofolo||64 385|
|Mmi Clinical Research Development Ireland (left the project)||106 830|
|Region Stockholm (left the project)||128 226|
|Universita Degli Studi Di Padova||20 000|
|Universita Degli Studi Di Roma Tor Vergata||650 000|
|Vseobecna Fakultni Nemocnice V Praze||151 035|
|Total Cost||67 000 001|