Lottie and Ashley

The future of children's research

The challenge

Although clinical trials have been a key part of health research for many years, it is only in the past decade that there has been increased focus on specific clinical trials for children’s medicines. Since 2009, the number of children taking part in clinical trials per year across the UK has more than doubled.

NIHR Clinical Research Facilities provide dedicated, purpose built facilities where cutting-edge clinical trials can take place. We take a look at how our facilities have supported the growth in clinical trials for children’s medicines.

The research

More than 4000 clinical research studies take place in year in NIHR Clinical Research Facilities, with six of our facilities specialising in children’s research.

This film series, led by Great Ormond Street Hospital (GOSH), highlights how pioneering clinical trials in NIHR Clinical Research Facilities are helping develop new treatments and cures for children with health conditions across the world.

The six films explore how pioneering novel therapies, precision medicine and innovative technologies are being used in paediatric clinical trials to help bring discoveries made in the lab to the patients who need them - from those affected by ultra-rare diseases through to common conditions that affect thousands of children. The films also show how collaboration across paediatric research centres has increased the number and complexity of children’s research studies and led to more opportunities for children take part in ground-breaking research trials.

The series features babies, children and teenagers who have all taken part in research trials at NIHR Clinical Research Facilities across England. The support of young people and parents is integral to paediatric research - this is reflected in the films, where young people or their parents are featured in conversation with researchers, sharing their own views on clinical trials and asking questions about children’s research.

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NIHR Clinical Research Facilities have enabled us to bring first-in-child trials and high intensity research to young patients and we are now seeing how this research has changed clinical practice and improved treatments for patients.

- Dr William van’t Hoff

Patient story

Lottie 14, and her younger brother Ashley, aged 12, have a rare form of rickets called X-linked hypophosphatemia (XLH). XLH is a genetic condition where the kidneys lose too much phosphate, which would otherwise help to strengthen bones. Children suffer bone pain, weakness and bending of the legs (rickets) and have poor growth.

Lottie and Ashley took part in a new clinical trial for children with XLH at the NIHR GOSH Clinical Research Facility. At the facility they receive an injection of the new drug that aims to maintain the levels of phosphate in their blood and improve their rickets.

Both Lottie and Ashley are keen to help others with their condition. Lottie said: “I joined the trial to help me and my family with my genetic condition.” Ashley added: “My parents told me it could help other people with our condition and it would help my family too, lots of us have XLH.”