Inside the Clinical Research Facility

Walk down to Level 1 of the Frontage Building, and you'll discover the National Institute for Health Research (NIHR) Great Ormond Street Clinical Research Facility (CRF). This bright and colourful purpose-built centre provides a specialist ward area for children taking part in research studies.

“The CRF exists to provide expert research care and support to patients and families who have volunteered to take part in research, as well as guiding researchers and companies who bring their studies to Great Ormond Street Hospital (GOSH)”, says Director of the facility, Dr William van't Hoff.

"We support and carry out all sorts of research studies," William explains. "From Phase 1 clinical trials in which new agents are tested in a small number of patients for safety, to studies of new diagnostic tests, genomics and imaging.”

Earlier this year the CRF was awarded £3 million of funding in a competitive funding scheme from the NIHR to help support its work over the next five years.

“This award reflects the team’s expertise in delivering complex, early-phase clinical research and makes us part of an internationally recognised, UK-wide network of clinical research facilities,” says William.

Key to the success of the centre is its research-trained staff. This includes research nurses whose expertise span all clinical areas, a trials pharmacy team who prepare the drugs and treatments, and a dedicated play specialist.

"We also have a fantastic team of managerial, administrative and laboratory staff who make sure all our research trials are carried out in a safe, controlled setting and to the highest scientific standards," adds William.

Because GOSH is a research hospital with trials taking place in all clinical areas, CRF staff help to support studies taking place on other wards across the hospital, as well as those taking place within the CRF. Two research advisory groups, made up of parents, carers and young people, help researchers and the CRF by commenting on research studies and the information about them.

Since the CRF was established in 2008, it has helped bring many new treatments from the bench to the bedside. William says: “One recent example is of nusinersen, a new drug for infants with a severe form of spinal muscular atrophy that was found to be effective in a CRF research trial led by Professor Francesco Muntoni. Nusinersen has recently been approved in the US and Europe and is now being used to treat GOSH patients. This shows how important clinical research is in bringing new treatments and cures to children with complex, life-limiting conditions.”

“My specialty is novel therapies research trials, which often require very complex treatments like enzyme replacement therapies to be administered. Working on novel therapies is really rewarding as there is often no established treatment for some of the patients at the CRF. The cutting-edge therapies that are used in research studies give these patients the chance to improve their condition.

"I’ve worked at the CRF for nearly two years now so I have got to know lots of the patients and families who are on longer-term trials. Recently, a patient on a long-term research trial drew a lovely picture of all the members of the CRF team who had cared for her. It’s now pinned on the noticeboard in the CRF staffroom! That was a special moment!”

Learn more about research at GOSH, by visiting our research information webpages.