What does 'Clinical Trial' mean to you?

A clinical trial is the last step of rigorous research testing that a new treatment must go through before it can be considered for everyday use against a particular condition. It can be using a new treatment or repurposing an existin drug and it can involve patients, healthy people, or both.

Due to the complex nature of the conditions that we treat at GOSH, for many of our patients, a clinical trial is their only hope of a treatment or cure.

We have over 200 clinical trials each year, and many of our 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 clinical trial PHYOX2 (DCR-PHXC-201) is a double-blind, randomized, placebo-controlled trial in around 36 patients with primary hyperoxaluria (PH).

Primary hyperoxaluria (PH) is a group of rare genetic metabolic disorders where a substance known as oxalate builds up in the kidneys and other organs of the body. It’s caused by a malfunctioning enzyme that normally prevents this build-up.

Children with PH have recurrent kidney stones and will eventually need dialysis or even a kidney transplant. The two-year PHYOX2 trial will study if nedosiran can keep oxalate levels at a normal level, limiting the formation of kidney stones and the damage that requires an eventual transplant.

Laura Chiverton and Hamza Meghari are part of the team behind the PHYOX2 trial at GOSH.

Laura did her nursing degree at Keele University and started off as neonatal nurse, caring for premature and sick babies at St Mary’s Hospital in Manchester. She moved to GOSH in 2019 as a Research Nurse in the CRF.

“My role in research and clinical trials is really varied. I run the study visits, discuss and arrange visits from sponsors, bring patients we have recruited into hospital, checking what’s needed in their trial protocol and what samples they may need to give us.

“Usually the patient’s family will discuss the trail with them and if they are broadly interested, I will discuss the research in more detail and what’s required from them in terms of extra samples and visits. Even if they agree, they have to go through a ‘pre-screening’ step so not everyone can actually be on the trial. Some trials are made extra tough as they have particular exclusion criteria that rules out a lot of patients.

After I’ve run the visit and entered all the data, it’s over to Hamza to ‘tidy things up’ and get that crucial data to the sponsor.”

Hamza Meghari got his BSC in nursing at Palestine College of Nursing and then worked in Jordan as a diabetic foot nurse, and came to UK in late 2017 to do a Masters degree in Global Health and Development at UCL. While at UCL, he learnt about some of the exciting clinical research at GOSH and wanted to get involved. He joined the team in the PICU in 2019 and then joined the CRF as a Research Co-ordinator in 2020.

“GOSH is a world-leading children’s hospital and I can’t think of a better place to be involved in research – it makes me so happy to see patients benefit from the work we do.”

Laura

“Starting the trial in the middle of the pandemic was difficult because the sponsor couldn’t come to site, we had fewer patients coming and less opportunities to discuss consent and then patients didn’t want to come into central London when travel was limited. We didn’t start that late though and got underway just a few months later than planned in the Summer of 2020.”

Hamza

“The pandemic definitely impacted our recruitment and even things like equipment delivery slowed down but we are getting back to normal - and the pandemic allowed us to try new things.

“We rolled out home-dosing so patients didn’t always need to come to GOSH and we started doing virtual visits, a new experience for us but one I’m sure we will build on. We couldn’t have done any of this without the work that was put into developing EPIC and GOSHLink to improve how we could input and access research data, so we owe a huge thanks to those teams.”

Laura

“Clinical trials mean giving families and patients that extra hope when other options are limited. They can change lives and I’ve seen clear examples where it’s made a specific difference and life altering changes. I really like being at the forefront of medicine – that’s what a clinical trial is.

Hamza

“For me clinical trials are the only way we can investigate new treatments for complex problems – they are invaluable. It’s in real patients and we can find real answers for them. I am proud playing a more active role in healthcare and scientific discovery. I believe my contribution could help future

generations have healthier lives.”

Find out more about our Clinical Research Facility