In one of the most severe congenital heart defects, hypoplastic left heart syndrome (HLHS), the left ventricle is underdeveloped and the prognosis is worse than in most other heart defects. The underdevelopment can occur gradually during fetal growth caused by a narrowing of the aortic valve. At some international centers, such fetuses are treated with a balloon dilation of the narrowed valve, but there is no scientifically sound evidence that this treatment is effective. The aim of this study is: 1/ ...

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Type 1 diabetes (T1D) is a chronic condition of childhood, affecting 1 in 490 children under the age of 15 years. It is caused by the immune system damaging the pancreas, the organ which makes insulin, and impacts every aspect of a person’s life. T1D has recognised stages before symptoms develop, providing an opportunity for early diagnosis, education and treatment which may delay the onset of symptoms. Early diagnosis often relies on a test called the oral glucose tolerance test ...

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Glioblastoma multiforme is a type of brain tumour. When it is first diagnosed,patients are usually treated with surgery and then a combination of radiotherapy and chemotherapy with temozolomide. Unfortunately,although this often slows or stops the disease from growing for a period of time,in most cases,usually a few months after the end of the original treatment,the tumour starts to grow again. This can be detected by an MRI scan. When this happens patients may experience new symptoms or a repeat of ...

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The purpose of this study is to investigate possible causes of nerve damage in patients carrying a genetic abnormality in the gene known as C9orf72, which is the cause of the diseases Amyotrophic Lateral Sclerosis (ALS; also known as motor neuron disease or MND) and Frontotemporal Dementia (FTD) in some people with these conditions. To carry out the study, we will take samples from and perform scans of the nervous system of carriers of the C9orf72 gene abnormality who have ...

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Mantle cell lymphoma (MC) is an uncommon type of non-Hodgkins lymphoma. Most patients with MCL need to stat treatment soon after diagnosis,usually with a combination of chemotherapy and a 'targeted' treatment called rituximab. 25-30% of patients have 'indolent' (non-aggressive) MCL where the standard approach is an initial period of 'watchful waiting' followed by treatment with rituximab and chemotherapy when the lymphoma worsens (progresses). In this trial we will investigate whether there is a benefit of giving earlier treatment with ...

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Resistance to standard of care treatment for stage III unresectable or stage IV melanoma can develop after about 12-15 months. During this period,the treatment will kill the less resistant cells,meaning a tumour has a greater proportion of cells that are resistant to treatment. This study aims to investigate if the patient having breaks in their treatment,guided by the ctDNA TAB level,allows the less resistant cells to continue to grow,resulting in a tumour with a lower proportion of resistant cells,making the ...

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Eczema is a common and long-term condition, which affects 20% of children. It is characterised by dry, itchy and inflamed skin. Symptoms tend to come and go, and there are many reasons why a worsening or “flare” of eczema can happen. Many parents wonder whether a food allergy might be a cause, however there is currently no good research evidence to support this. In the TIGER study, we want to find out whether making changes to the diet of ...

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The Staphylococcus aureus network adaptive platform (SNAP) trial investigates a range of antibiotic treatment options in S. aureus bacteraemia (SAB). Background: S. Aureus bloodstream infection (bacteraemia) is a common,severe global infection. SAB affects ~13000 people/year in England with 15-30% 90-day mortality. Treatment is based on antibiotic susceptibility but we lack evidence to support antibiotic selection with <3000 participants ever recruited into randomised controlled trials (RCT). There are no specific SAB international or UK guidelines. Pragmatic clinical trials are urgently needed,to optimise antibiotic choice ...

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The ADVANCE study is post-market, prospective, interventional, global, multicenter, randomized, dual-arm trial. The purpose of the post-market study is to demonstrate clinical benefit with Endurant II/IIs compared to Gore Excluder/Excluder Conformable by generating robust, Level I evidence to elucidate the mechanism of sac regression and any device-related differences, in subjects with Abdominal Aortic Aneurysms (AAA) for whom an EVAR procedure is indicated. Beyond comparing sac regression rates between devices, the study will also investigate if the sac regression led to decreased risk ...

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Retinal degeneration has a very significant impact on quality of life. For example, age-related macular degeneration (AMD) is one of the leading causes of vision impairment worldwide. AMD involves progressive damage to the central part of the retina (the tissue at the back of the eye that detects light) called the macula, which is responsible for our central vision and functions such as reading and seeing faces or fine detail. Treatment is currently available for one type of AMD called ...

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