Respiratory distress syndrome (RDS) is a condition commonly found in premature babies that causes breathing difficulty soon after birth. Many babies respond to breathing support alone, but some need surfactant, a liquid drug given into the baby’s airway. Traditionally, the standard method to administer surfactant is through a tube inserted into the airway by the treating doctor. The doctor first needs to visualise the baby’s airway using a laryngoscope (a metal device that holds the airway open), placed into the baby’s mouth and positioned correctly. ...

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The main purpose of this study is to develop measures that capture how Huntington's disease (HD) affects an individual's health and well-being, specifically during the stage of the disease when people have had signs and symptoms for several years. It will also be important to see whether these measures can be completed by the companion participant (either in-person or remotely, e.g. phone) on the HD participant's behalf. The study is made up of two parts. Part 1 will evaluate a modified questionnaire called the ...

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Patients with diabetes have a higher risk of heart attack and worse outcomes. While high glucose levels seem to drive this risk, glucose-lowering medicines do not reduce the risk. Work by Professor Choudhury’s group has shown that high glucose levels lead to re-programming of a type of white blood cell involved in processes such as inflammation, that lead to narrowing of the blood vessels in the heart and consequently heart attack. Specifically, high glucose makes cells in the bone marrow ...

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This is a phase 1b vaccine efficacy study of ID93/GLA-SE in historically BCG-vaccinated and BCG-naive healthy adult volunteers using an aerosol BCG challenge model. There will be four study groups with twelve volunteers in each group (Table 2). Volunteers will be randomised to eligible groups based upon their BCG vaccine status,and will either be given 2 μg ID93 + 5 μg GLA-SE at Day 0 and Day 56 (Group A and C) prior to BCG aerosol challenge,or proceed directly to ...

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von Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population. Despite this, little is known about its natural history, or of the impact it has on affected individuals. The Haemnet vWD.360 programme is a mixed-methods, natural history study designed to better understand vWD and its impact on individuals. It comprises both qualitative and quantitative approaches and is designed to include the perspectives of individuals with a diagnosis of ...

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Primary Aim: 1. To assess the bias,precision,limits of agreement and 30% accuracy of eGFR equations compared to measured GFR for people of different ethnicities (e.g. South Asian,Chinese,South-East Asian and Black) in the United Kingdom,compared to people of reported White ethnicity. Design: Retrospective observational data only multi-centre cohort study. Study Population: People with both normal kidney function and chronic kidney disease,who have had a measured GFR test and a paired eGFR test (within 30 days),between 2009 and 2022 inclusively. The study population will include ...

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This is a post authorization safety study of patients treated with fenfluramine hydrochloride oral solution, conducted at centers prescribing fenfluramine in routine practice. This study will involve neuro-pediatrician's, pediatricians, or neurologists – with experience in the treatment of epilepsy in both European (Germany, Austria, Denmark, Italy, UK, Spain and France) and non-European countries (potentially including US and Japan). The study will last for 10years for first patient first visit with enrollment period spanning over 3years from registry start date. The maximum ...

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This is a phase 1 study exploring differences between the immunological responses to revaccination with intradermal-BCG and aerosol-inhaled BCG in healthy adults and those with pre-existing T2DM. There will be three study groups with twelve volunteers in each group (Table 2). Volunteers will be enrolled into groups based upon whether they have a diagnosis of T2DM. They will then be vaccinated with intradermal BCG (Groups A and C) or aerosolised BCG (Group B). Objectives: Co-Primary: To compare the immunological response of BCG ...

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In patients with heart failure, serum hypomagnesaemia is common and attributed to increased renal magnesium (Mg2+) secretion and diuretic therapy. Serum hypomagnesemia is associated with increased heart failure presentations and hospitalisations in patients with heart failure with preserved ejection fraction (HFpEF). There is a poor correlation between serum Mg2+ (the compartment which can be measured) and intracellular Mg2+ concentrations9 (the compartment in which Mg2+ regulates cellular processes) with serum Mg2+ being a poor surrogate for total body Mg2+ content. Using ...

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ARTEMIS is a phase II,multi-centre,open-label,randomised controlled trial using IMRT/VMAT/TomoTherapy,comparing radiotherapy (SCRT/LCCRT) followed by chemotherapy (FOLFOX/CAPOX),with or without the addition of AN0025 throughout treatment. Eligible patients will have moderate to high-risk rectal cancer where pre-operative chemoradiotherapy (CRT) or total neoadjuvant treatment (TNT) are standard treatment options and who are interested in organ preservation. ARTEMIS uses the Sargent’s three-outcome two-stage comparative design,including an interim analysis for futility. The trial will randomise 140 patients,from 15-20 UK radiotherapy sites,with a 1:1 allocation ratio to receive ...

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