c4c

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.

Find out more

• Read the article in full
• If you're interested in getting involved in the project, you can sign up to the c4c patient database.

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.

Find out more

• Read the paper in Nature Reviews Drug Discovery
• Read the guidebook entries on c4c, EUPATI, and PREFER
• Orphan Drug Development Guidebook Materials on the IRDiRC website

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.

Read more

• IMI’s news story
• c4c project website

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.