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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Patient-reported outcome measures (PROMs) are questionnaires that measure aspects of somebody’s health, like hand function. They can monitor a person’s health over time. Our team has made a smartphone app that make these questionnaires shorter. Previous research suggests that unlike the full-length questionnaire, people do not mind using the app every day. This could show us how quickly people recover after different types of hand surgery. We want to see whether our app could be used to compare patients’ experiences ...

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We may be able to rescue a generation of patients who are being abandoned to progressive injury to their heart from previous-generation pacemakers. This is because there is no randomised evidence to show that they would fare better with upgrade to modern pacing strategies. Today, if patients who require pacemakers for slow heart rates have even mildly impaired heart function, they receive modern pacing strategies (via His bundle, left bundle or biventricular pacing) rather than traditional right ventricular (RV pacing. ...

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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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Raised blood pressure (BP) during and following pregnancy affects around 80,000 women each year in the UK, and can lead to very serious problems such as pre-eclampsia or stroke. After birth, a women’s BP can remain elevated, but in most cases returns to normal over 2–12 weeks. During this period, medication needs to be adjusted to achieve the correct control. Research suggests that better BP control during this period is associated with improved long-term health outcomes. This trial aims to ...

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