This is a first-in-human, randomised (treatment is assigned by chance), study to evaluate the safety, tolerability and the pharmacokinetics [how a drug is processed] (PK) of multiple doses of the study drug (BIIB101) in adults with Multiple System Atrophy (MSA). Participants will receive either the study drug or a placebo (medication made of an inactive material). The study drug has been tested in animals but, since the study drug has not yet been tested in humans, the safety in ...

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Aim: To establish laboratory tests for measuring the composition of human breast milk Outline of research: This study involves the collection of 1-2 teaspoons of breast milk (up to 10 mls) from up to 30 women who are already expressing milk for their babies in the Oxford Newborn Care Unit, Women’s Centre, John Radcliffe Hospital, Oxford The women are healthy volunteers and are recruited by virtue that their babies are NHS patients. Duration of participant involvement will be around 45 min. ...

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CCS1477 is a new experimental medication (sponsored by CellCentric Ltd) for a group of cancers that effect the blood and/or bone marrow. These include Acute Myeloid Leukaemia (AML), high-risk Myelodysplastic Syndrome (MDS), Multiple Myeloma (MM) and Non-Hodgkin Lymphoma (NHL). It is aimed at tumours that are not responsive to, or have become resistant to, existing medications used in late stage disease. The purpose of this study is to examine the safety, tolerability, pharmacokinetics (PK) and efficacy of CCS1477 when treating patients ...

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PURA Syndrome was first described in 2014 in a small cohort of children, and since then just over 250 individuals have been identified to have the condition internationally. PURA syndrome typically causes severe developmental delay and intellectual disability. Many also suffer with hypotonia (severe floppiness), feeding difficulties and breathing abnormalities in infancy, and many develop seizures in early childhood. We are designing a longitudinal natural history study, with the aim to collect medical data on individuals with PURA Syndrome. We ...

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Hepatocellular carcinoma (HCC) and cholangiocarcinoma are the two most common causes of primary liver cancer and HCC is the second highest cause of cancer death worldwide. It is known that most of these cancers occur in patients who already have a liver condition. Despite close monitoring of many patients who have liver disease with regular ultrasound scans, HCC and cholangiocarcinoma are often discovered at a late stage. This is because they rarely cause symptoms until they have ...

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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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