Sheffield Children’s NHS Foundation Trust has become the first site in Europe to recruit a patient to a major international clinical trial investigating a potential new treatment for a severe form of epilepsy – Dravet syndrome.
The National Institute for Health and Care Research (NIHR) Sheffield Children’s Commercial Research Delivery Centre (CRDC) is one of the sites delivering the study that will see up to 150 participants take part across Europe, the United States and Japan.
Dravet syndrome is a severe form of epilepsy, which often begins before the age of one and affects about one in every 15,000 babies born in the UK.
Three-year-old Penny travels from her home in Blackpool, Lancashire for specialist care here at Sheffield Children’s. Her mum Roberta said: “Penny is the most determined little girl, and we are so proud of her. We are so grateful to be able to take part in a trial that may bring us a step closer to finding a better treatment for Penny and all the other children with Dravet syndrome.
“The whole team at Sheffield Children’s has been amazing and it has been mind-blowing to be surrounded by such an experienced and understanding team.”
Gillian Gatenby, Associate Director of Research at Sheffield Children’s and Deputy Director of the NIHR Sheffield Children’s CRDC, said: “At Sheffield Children’s, research isn’t a bolt – on, it’s embedded in how we provide care and improve lives. Recruiting the first European patient to this trial embodies that ethos and is a fantastic achievement for our research team.
“As the only CRDC dedicated to delivering commercial clinical trials for children and young people, we are committed to bringing more cutting-edge treatments and therapies to children like Penny, ensuring that children from our region and all across the UK have greater access to life-changing research opportunities.
NIHR Sheffield Children’s CRDC is part of the CRDC UK Network, which brings together 35 centres across primary, secondary and specialist care to strengthen the UK’s position as a global leader in commercial clinical research.
Supported by the NIHR in partnership with the pharmaceutical industry, the Network provides a coordinated UK‑wide infrastructure for fast, reliable and consistent trial delivery at scale. It offers industry access to high‑performing research sites and enables inclusive, decentralised trials, allowing more people from more communities across the NHS to take part in research.
Professor Melanie Davies, Director of the CRDC UK Network, said: “Congratulations to the team at the NIHR Sheffield Children’s CRDC for recruiting the first participant in Europe to this important trial. This is a significant milestone and a testament to the dedication and expertise of the team involved.
“The CRDCs were created to accelerate life‑changing research across the UK, and this achievement highlights the impact they are already making. None of this progress would be possible without people who give their time to take part in research, and we are incredibly grateful to participants like Penny whose involvement helps drive the development of new treatments.”
As Sheffield Children’s marks its 150th year, the Trust is continuing to build on a proud history of research and innovation, helping to develop the treatments and discoveries that will shape the future of children’s healthcare.
You can find out more about research and innovation at Sheffield Children’s by visiting: www.sheffieldchildrens.nhs.uk/research