We are conducting a study to gather more information on the safety and effectiveness of Xromi (hydroxyurea), a liquid medicine licensed to treat sickle cell disease (SCD) in children from 9 months of age. We aim to understand its safety profile and efficacy in preventing serious SCD complications (e.g., pain crises, acute chest syndromes, stroke) in children under 2 years when used in standard care. We will compare medical information collected during routine clinical appointments of children under 2 years who ...

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AVEIR LP UK Registry is a study collecting data from patients who need to have a leadless pacemaker inserted due to underlying heart diseases. Leadless pacemaker technology promises to revolutionise care of the patients that require pacing. The AVEIR pacemaker is the newest of approved leadless pacemakers. Vigilant surveillance of its use in real clinical practice with respect to the effectiveness and safety of the device, at the time of the procedure as well as in the long-term, is essential ...

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Patients with chronic obstructive pulmonary disease-obstructive sleep apnoea (COPD-OSA) overlap syndrome have higher rates of COPD exacerbations compared to patients with similar severity COPD without OSA. It is currently not known whether treating OSA in those with COPD-OSA overlap reduces exacerbation rates. COPD exacerbations are characterised by acute transient worsening of symptoms such as dyspnoea, sputum production, sputum purulence and cough which are above the normal day to day variation in symptom burden and are usually associated with escalation of ...

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The purpose of this study is to measure the PK, PD, safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to < 18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive 4 once-weekly injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of ...

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Despite the lack of evidence on safety or efficacy, many clinicians offer whole body therapeutic hypothermia to infants with mild hypoxic ischaemic encephalopathy, and even to those without any encephalopathy. Such a wide variation in clinical practice within the NHS not only leads to poorer outcomes and may harm infants who would have otherwise done well, but masks improvements in obstetric care and increase litigations. The COMET trial will establish the safety and efficacy of whole-body hypothermia for mild hypoxic ischaemic ...

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Myeloma (MM) is an incurable blood cancer. While survival has substantially improved over the last decade, there is still huge variability in clinical outcomes between patients. The current approach to drug development assumes that all patients respond similarly to a given drug despite the fact that only a third of MM patients are eligible to take part in therapeutic clinical trials. Within the NHS, the current treatment approach to MM means that all patients receive the same treatments according ...

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The main purpose of the trial is to determine when the most beneficial time for people to start or restart a DOAC after their head injury is. People will be asked to start the medication either 1 week or 4 weeks after their head injury. This will be randomly assigned by a computer. They will then be followed closely for 12 weeks and any major bleeding events or a blood clots (thrombotic events) such as a stroke or heart attack ...

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This is a Phase 2a, randomized, double-blind, placebo-controlled study to evaluate the efficacy, tolerability, and safety of frexalimab, SAR442970, and rilzabrutinib in participants from 16 to 75 years of age with biopsy-proven primary FSGS or MCD, with eGFR ≥ 45 mL/min/1.73 m2, and UPCR ≥ 3 g/g at screening, who have demonstrated at least partial response (≥ 40% UPCR reduction) to at least one previous immunosuppressive therapy. The purpose of this study is to measure the change in proteinuria and its impact ...

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Transcatheter aortic valve implantation (TAVI) is a key-hole technique to replace a faulty (narrowed and/or leaking) aortic heart valve. The replacement aortic valve is delivered to the heart through tubes inserted in a main artery (usually the artery in the groin). Over the past 15 years TAVI has rapidly grown and has taken over from open-heart surgery (surgical aortic valve replacement (SAVR)) to become the most commonly used technique for aortic heart valve replacement. Although TAVI is a safe and effective ...

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Background Sciatica is a term used to describe pain in the leg, but it is actually caused by an irritation of the small nerves in the lower back. One in four people with sciatica also report weakness in the affected leg. In my recent work, 85% of people coming to a hospital appointment for sciatica reported leg weakness, but our hospital tests detected weakness in only 34% of them. This means that many people have a problem that their health ...

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