This is a FTiH modular, Phase I/II, open-label, multicentre, dose escalation and expansion study in participants with r/r haematologic malignancies. The study consists of several study modules, each evaluating the safety, tolerability, PK, pharmacodynamics, and efficacy of orally administered AZD3470 as monotherapy and in combination with other anticancer agents. This study will follow a modular protocol design evaluating AZD3470 as monotherapy and in combination with other anticancer agents. New cohorts (including further monotherapy expansion) and new modules for combination treatments may be ...

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Childhood and adolescent cancers comprise a heterogeneous group of rare diseases, mostly distinct to those diagnosed in adults. Survival of children and adolescents with cancer has considerably improved in recent decades, but cancer remains the most common cause of disease-related mortality in developed countries. In the case of disease relapse, the prognosis for paediatric patients is poor with survival rates of < 20%. Better understanding of tumor biology has led to exponentially increased development of novel targeted therapies and immunotherapies in ...

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Nemaline myopathies(NM) are genetically determined conditions that usually present with muscle weakness, breathing problems and difficultly feeding. The severity of NM varies from significant muscle weakness requiring a wheelchair, breathing and feeding support with signifcantly reduced life expectancy to milder forms in which the person remains able to walk and has a normal lifespan. Current treatments for people living with NM are supportive only. Several potential therapies are in development which may be available in the next 5-10 years. ...

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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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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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