People with familial hypercholesterolaemia (FH) have raised cholesterol levels in their blood from birth. Cholesterol is a type of fat our bodies need but having raised levels over a long time causes heart disease, such as heart attacks. To prevent future heart disease, guidelines recommend starting cholesterol-reducing medication, usually statins, by age 10. However, there remain unanswered questions about what is the right age and cholesterol level at which to start statins to prevent later life heart disease. Considerations are: avoiding ...

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This multicenter, multinational, natural history study is designed to retrospectively and prospectively collect clinical data in participants with genetically confirmed DOK7-CMS. No IMP will be administered. Participants will attend up to 3 study visits and complete home digital physical activity monitoring for 1 week after each visit. The assessments will evaluate participants’ symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design. Approximately 30 male and female participants ≥18 years of age ...

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Multisite,multi-arm,open-label,pragmatic randomised controlled trial with integral internal pilot and concurrent economic evaluation. The trial is predicated on two primary comparisons of (1) a monotherapy (i.e. Iron) and (2) a combination therapy (i.e. Iron + EPO),compared with a single common control group (usual care) respectively,in a superiority hypothesis testing framework.

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This is a Phase 3, randomized, controlled, multicenter, open-label study to evaluate the efficacy of IMC-F106C + nivolumab compared with nivolumab regimens in HLA-A*02:01-positive adult participants with previously untreated advanced (unresectable or metastatic) melanoma. Participants will be randomized 1:1:1 to IMC-F106C 40 mcg + nivolumab (Arm A), IMC-F106C 160 mcg + nivolumab (Arm B), or a nivolumab regimen (Arm C). The control Arm C will be country-specific and include: • Nivolumab + relatlimab, an emerging SoC, in countries where this combination ...

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cases in 2020 globally (11.7% of all new cancers). Breast cancer is also the fifth most common cause of death from cancer, with an estimated 685000 deaths in 2020 (Sung et al 2021). Despite advances in the diagnosis and treatment of breast cancer, around 5% to 10% of women diagnosed with breast cancer have metastatic disease at the time of diagnosis, with a much higher percentage in low- and middle-income countries, and up to 30% of women with early-stage non-metastatic ...

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Each year in England, about 3,000 babies are born with one of about 200 genetic conditions that are treatable in early childhood. Nine of these conditions are currently looked for in the NHS newborn bloodspot test. But there are many more conditions where earlier diagnosis and treatment could help. This study investigates the genomes of newborn babies to see if we can find and treat rare genetic conditions early. A genome is a person’s entire genetic sequence ...

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MAT is an international,multicenter,prospective,parallel-group,randomised,double-blind,placebo-controlled trial. The double-blind period will be preceded by a 6-week,single-blind (participants will remain blind),active run-in phase in which all potential participants will receive metformin sustained release (SR) with fortnightly dose increments. The aim of this run-in phase is to ensure the randomisation of participants who tolerate the treatment regimen and to increase the likelihood of high treatment adherence during long term follow-up. Randomised participants will receive study medication for an average of 3.5 years (up to ...

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Cirrhosis, progressive scaring of the liver- has many causes, principally, excessive alcohol intake, fatty-liver and viral infections. Unlike many chronic diseases, cirrhosis deaths are increasing rapidly year-on-year. It is the third commonest cause of premature, UK working-age deaths, with 62,000 years of working-life lost each year and NHS care costs of £4.53bn annually. One quarter of all UK cirrhosis patients are at-risk of acute decompensation, whereby complications such as fluid-overload, confusion and infections arise, requiring hospital-emergency treatment. Currently, decompensated cirrhosis patients ...

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Juvenile Idiopathic Arthritis (JIA) is a chronic inflammatory disease affecting 1 in 1000 children with around 12,000 children with JIA in England and Wales [1,2]. Delay in diagnosis and under-treatment lead to joint damage,disability and reduced quality of life [3]. In the UK and most of the world,children and young people with new-onset poly-articular Juvenile Idiopathic Arthritis (pcJIA) are commenced on corticosteroids and methotrexate [2,4,5]. As methotrexate can take several months to have an effect,corticosteroids are used to rapidly control ...

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Nearly 30,000 infants under one are admitted to hospitals in England each year with bronchiolitis, a common viral chest infection. Half of them need oxygen treatment through ‘nasal cannula’ (thin tubes inserted into the nostrils). Most infants respond to nasal cannula oxygen alone, but some need additional breathing support. In these infants, different treatments such as ‘humidified’ (moist) standard oxygen (HSO), high-flow nasal cannula (HFNC) and ‘CPAP’ (pressurised oxygen delivered through the nose) are used. However, it is not clear ...

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