The EXPERTS-ALS platform is designed as a randomised,open-label,multi-arm trial with a biomarker-based endpoint. It aims to prioritise drugs for further testing based on their ability to lower NFL levels and to also identify drug-specific adverse events.The EXPERTS-ALS protocol describes an overarching trial design to evaluate the effect of candidate drugs on blood NFL levels in patients with ALS receiving usual standard of care. The protocol is deliberately flexible,allowing: as broad a range of ALS patients to be recruited; participant randomisation ...

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This study will evaluate long-term safety and efficacy in approximately 400 subjects who were dosed with SRP-9001 in a previous clinical study. The study will consist of 2 periods as follows: • An Eligibility Confirmation Visit of up to 2 days in length, during which eligibility confirmation, informed consent, disease characteristics, and current therapy/ concomitant medication data will be collected. • A Long-Term Follow-Up Period that will begin the day eligibility is confirmed and continue for at least 5 years post-infusion of ...

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The purpose of this study is to investigate if it is worthwhile prescribing medicines to reduce blood clots after varicose vein procedures. People enrolled in the study will undergo an assessment to make sure that they don’t have the most important risk factors for clots. Those most at risk would not be included in the trial. People enrolled in the study will be over 18 years of age and would receive compression stockings along with one of the following three ...

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BACKGROUND Venous thromboembolism (VTE) is a major global health burden. Many people who survive VTE suffer from longer term physical complications or psychological sequelae. Temporary lower limb immobilisation (plaster cast,walking boot or rigid splint) and injury are combined risk factors for VTE. Approximately 70,000 people are immobilised after injury every year in the UK. VTE in this setting is potentially preventable through early prophylaxis with anticoagulant medication. However,baseline VTE risk varies widely across the population and there are multiple drugs for ...

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This is a multicenter RWE study to evaluate the efficacy of etrasimod daily dose of 2 mg in patients with moderately to severely active UC. The study consists of a 52-Week treatment period, and a 28-day safety follow-up period. This prospective, international, non-interventional primary data collection study will be conducted in countries across the world. Countries will be divided into study regions. A country can be a study region on its own. Each study region will start with patient recruitment as soon ...

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What is the problem? Skin cancers are common and the number of people who are developing them is increasing. They are usually split into two groups: melanomas are less common but more serious and keratinocyte skin cancers are more common but less serious. Most need to be cut out. Keratinocyte skin cancers on the leg often cannot be closed with stitches and the wounds are left open to heal by themselves (known as secondary intention wound healing). When left open, they ...

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The overall aim of this research is utilise blood donated from patients on warfarin therapy to aid the development and optimisation of Microvisk’s second generation Prothrombin/International Normalized Ratio (PT/INR) Test System. The donated blood will be used on ongoing development, validation, verification, and calibration projects. Microvisk has developed a small hand-held device which will measure PT/INR from a low volume capillary blood sample. The device is intended for use by both healthcare professionals and for self-testing by the patient. Consenting patients of ...

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Allogeneic hematopoietic cell transplantation (alloHCT) is a well-established therapy for different lifethreatening hematologic malignancies. The use of alloHCT is constantly increasing, with nearly 20 000 transplantations reported to the European Society for Blood and Marrow Transplantation (EBMT) per year. However, this treatment is limited by high morbidity and mortality, mainly related to relapse, infection, graft-versus-host disease (GvHD), and conditioning-related toxicity. Therefore, overall survival (OS) and GvHD-free, relapse-free survival (GRFS) after alloHCT, remain low, around 60% and 30% at one year. Over ...

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Although the optimal treatment strategy in oesophageal (food pipe) cancer has been the subject of research for several decades, overall outcomes are very poor, with a 5-year survival of 15%. In the UK there are 9100 new cases of oesophago-gastric cancer diagnosed and approximately 7925 deaths every year (Cancer Research UK Oct 2019). Oesophageal cancer is a strategic priority for CRUK and a serious health problem world-wide. Pre-surgery treatment consists of chemotherapy or chemoradiation, but the optimal treatment is unknown. ...

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This placebo-controlled, double-blind, randomized, Phase 3 study is being conducted in adult participants with high-risk atherosclerotic cardiovascular disease (ASCVD) who are not adequately controlled by their maximally tolerated lipid-modifying therapy, to assess the impact of the obicetrapib 10 mg + ezetimibe 10 mg FDC therapy on coronary plaque and inflammation characteristics, evaluated using cardiovascular computed tomography angiography (CCTA). Approximately 300 eligible participants will be randomized in a 1:1 ratio, respectively, to the following treatment groups: • Obicetrapib 10 mg + ezetimibe 10 ...

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