Matei's story

20 May 2019, noon

Matei and Flavia

To mark International Clinical Trials Day 2019, we took a visit to the Clinical Research Facility to meet with one GOSH mum, Flavia, whose son, Matei, has been on a clinical trial at the hospital for the last couple of years.

Over 200 clinical trials are carried out at Great Ormond Street Hospital (GOSH) every year. By comparing the effect of one treatment against another, clinical trials help the hospital develop better, kinder medicines and improve the lives of children with rare and complex conditions.

At GOSH, many patients on clinical trials are cared for at the National Institute for Health Research (NIHR) GOSH Clinical Research Facility (CRF) – a specialist ward for children and young people who are taking part in clinical research studies.

The NIHR CRF was established with support from JN and Dame Phyllis Somers via GOSH Charity in 2008. GOSH Charity continues to support clinical trials at GOSH by funding vital roles like Research Nurses and Trial Coordinators.

Meet Matei 

Five-year-old Matei has a serious condition called Sanfillipo syndrome, which causes delays in development.

Matei was first diagnosed with the condition in 2016. His family were told that there was no treatment available, but that there was a new research trial that Dr Maureen Cleary at GOSH was about to start – trialing a treatment that aims to slow the condition’s progress.

The family were put in contact with the metabolic team at GOSH, and it was decided that Matei was eligible for the trial.

Visiting the hospital 

As part of the clinical trial, Matei visits the NIHR GOSH CRF every week where he is cared for by a multidisciplinary team, including: a Consultant, Senior Research Coordinator, Advanced Nurse Practitioner, Research Health Play Specialist and Assistant Clinical Research Practitioner – as well as many other clinical and non-clinical staff.

“They are very welcoming and help us a lot,” says Flavia. “Matei is always happy when we arrive at the hospital and runs to the entrance!”

Dr Maureen Cleary, Consultant in Paediatric Metabolic Medicine and lead researcher, set up the study a few years ago, and is responsible for recruiting patients to take part.

“Myself or another colleague sees Matei to check on his progress every week. The trial is very intensive so it’s a big commitment for Matei’s whole family - but they never fail to miss an appointment! The trial is truly multidisciplinary and relies on the expert knowledge of not just the CRF but from teams across the hospital – from psychology to cardiology, from pharmacy to ENT,” she says.

And where does play come into it?

“Play is important in supporting children and families to create an environment that feels safe and non-threatening,” explains Sian Spencer-Little, a Research Health Play Specialist funded by GOSH Charity as part of the hospital’s Play Team. “This can be through therapeutic play or offering specialised play opportunities to reduce anxieties. I also help young people engage and understand the role of research studies.

Play Specialist Sian

“Matei has a phenomenal personality. When he has his medication he is calm and knows just what activities he likes to help him. We usually sit together, singing and watching the iPad. This is distraction therapy at its best.“

The importance of research 

Through the clinical trial, Flavia feels that she’s learnt a lot about research. “I’ve learned a lot of things – I could almost be a nurse in the future!” she says, smiling.

“I think research is not only important for our family, it’s important for other families. In the future there will be other kids with different conditions or with the same condition as Matei – and maybe in the future there will be a treatment. When we enrolled Matei on this study, we were thinking about this – that maybe it could help other families.”

Flavia wants to raise awareness of rare conditions so that people have a better understanding.

To find out more about how GOSH Charity supports research, click here. 

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