FaR-RMS is an over-arching study for children and adults with newly diagnosed and relapsed rhabdomyosarcoma (RMS). It is a multi-arm, multi-stage format, involving several different trial questions. FaR-RMS is intended to be a rolling programme of research with new treatment arms being introduced dependant on emerging data and innovation. This study has multiple aims. It aims to evaluate the impact of new agent regimens in both newly diagnosed and relapsed RMS; whether changing the duration of maintenance therapy affects outcome; and whether changes to dose, extent ...

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Acute pancreatitis is a serious condition in which the pancreas becomes inflamed and is damaged. It causes intense abdominal pain and may lead to multiple organ failure. Those suffering from the disease may need help with breathing, heart and kidney function. One out of twenty patients with acute pancreatitis dies, which is more likely to happen to those who develop organ failure. Treatment can be prolonged, often with extended stays in hospital of many weeks. To date many drugs have ...

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Minimal Change Disease (MCD) and Focal Segmental Glomerulosclerosis (FSGS) are rare diseases that cause nephrosis. Patients suffer with debilitating oedema, and are at increased risk of infection and venous thromboembolism. Standard of care consists of high dose glucocorticoids, with associated morbidity including weight gain, diabetes, infection and osteoporosis. Patients require frequent hospital visits, hence these diseases carry a high socioeconomic burden. This is particularly high in FSGS, where patients with uncontrolled disease progress to end stage kidney disease (ESKD), at ...

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There are approximately 16,000 new cases of rectal cancer in the UK per year. Surgery is the mainstay of treatment which is associated with peri-operative mortality and long-term morbidity. Locally advanced disease is treated initially with preoperative radiotherapy, in the main using long-course chemo-radiotherapy (LCCRT) at 45 to 50 Gy, followed by major surgery 8 to 15 weeks later (referred to in this protocol as standard surgical pathway) or selectively by short-course radiotherapy (SCRT) at 25 Gy, traditionally followed by ...

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It is particularly difficult to treat some groups of children diagnosed with cancer, including very young infants in the first weeks of life and children with poor kidney function. It can be especially difficult to know how much chemotherapy (anti-cancer drugs) to give to these children. Doctors often have to make difficult decisions about the most appropriate dose of drug, without enough scientific information to help them decide. This can mean that patients might not get enough drug or get ...

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We address whether maladaptive learning systems contribute to the maintenance of chronic pain. This has been difficult to answer because learning comprises a set of complex,interacting processes,and hence difficult to evaluate and quantify. Based on computational models of learning,we have designed a suite of tasks and analysis tools that probe domaingeneral value-based and sensorimotor learning. These are implemented online as a set of tablet-based computer games that can be applied easily and widely in observational or longitudinal clinical studies in domestic settings. We will study ...

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This study aims to understand the molecular pathophysiology of sepsis in order to improve patient care. Sepsis is defined as life-threatening organ dysfunction caused by a dysregulated host response to infection. Currently we do not understand why only a minority of patients develop this extreme response to infection, why there is variability in the sepsis response and how best to identify specific sepsis patients who will benefit from particular treatments. This is important as in the UK, sepsis is a ...

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Our aim is to establish a Spinal Muscular Atrophy (SMA) National Platform to improve UK standards of care, manage National and International clinical trials and facilitate translational research for this common neuromuscular disease. To achieve this purpose we will start to systematically collect longitudinal validated outcome measures for SMA children followed at GOSH, the largest cohort followed in UK, and pilot and update novel outcome measures. This will be done ensuring that data collected are not only clinically meaningful but also robust for ...

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Prof Antoniades' research group has recently discovered a new way of analysing pictures of the heart using computed tomography (CT) scans. The new measure can identify people who are at risk of having a heart attack, years in advance. In order to introduce this CT measure into clinical care, it must be shown that it can be successfully measured in different CT scanners (in different hospitals) within an acceptable and pre-defined range. Therefore, the study is looking to recruit 90 ...

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The purpose of the research is to collect information from adult and children with eczema, starting certain treatments, referred to as ‘systemic immuno-modulators’. As eczema is often a long-term condition, it is important to establish how well these medicines work with regard to improvement in disease severity, quality of life and also safety, especially when these are used for longer periods of time. All medicines prescribed for eczema in clinics have already undergone careful testing in clinical trials, however, ...

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