There are about 25435 clinical studies being (or have been) conducted in United Kingdom. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases. In this study no therapy is being offered.
The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.
Iron deficiency is the most prevalent nutritional deficiency worldwide with one in four estimated to be affected by iron deficiency anaemia. Women of reproductive age are at greatest risk for iron deficiency and anaemia due to iron losses during menstruation and childbirth as well as the increased need for iron throughout pregnancy. However, iron deficiency without anaemia is at least twice as common as iron deficiency anaemia with females aged 11-49 at the biggest risk of all. Despite this, it is commonly left undiagnosed. Those who are iron deficient non-anaemic can still suffer from the same common consequences of iron deficiency anaemia; these include unexplained fatigue, mood changes and decreased cognitive performance. However, randomised controlled trials (RCTs) assessing the effect of iron supplementation upon cognitive performance, mood, fatigue and well-being in non-anaemic iron deficient women of reproductive age are limited. There is also a lack of well-defined diagnostic criteria for non-anaemic iron deficiency, which makes comparisons across RCTs difficult. However, there is evidence to suggest that a haemoglobin cut off of ≥120 g/L and serum ferritin ≤ 20 µg/L provides an accurate indication of non-anaemic iron deficiency in women of reproductive age; this is inclusive of the ability to recognise iron-associated deficits in psychological and physiological functioning. Additionally, previous RCTs could be improved by utilising a lower dose of iron in a bis-glycinate chelate form, which is evidenced to have superior bioavailability, tolerability and subsequent efficacy compared to ferrous formulations. Iron bis-glycinate absorption is also negatively associated to serum ferritin levels, which is suggestive of a non-anaemic iron deficient population benefitting most from it's administration. The current study aims to build upon previous iron RCTs in populations of non-anaemic iron deficient and iron sufficient women of reproductive age by investigating the effects of 16-weeks supplementation with either iron bis-glycinate chelate alone, iron bis-glycinate plus vitamin C (as ascorbic acid) or matched placebo upon cognitive performance, subjective mood, fatigue, health and well-being.
All patients with heart disease should have the opportunity to participate in research into their condition, to advance knowledge and treatment. The HeartHive COVID-19 study is an international online pilot observational cohort study evaluating the impact and clinical outcomes of the COVID-19 pandemic on subjects enrolled in the HeartHive. Cardiomyopathies are progressive diseases, and there is a need to better understand what factors affect the chances of developing cardiomyopathy, and how the condition progresses. The impact of the COVID-19 pandemic on patients with cardiomyopathy has not been explored and represents a critical unmet need. Insights into exposure, healthcare outcomes, behavioural changes and the psychosocial ramifications of the pandemic are required to better understand the health needs of this population during these unprecedented circumstances and to adapt clinical services to meet these. The study will entail completing serial online surveys during the pandemic. This study uses The Heart Hive - an international, online registry of patients with self-reported clinically diagnosed cardiomyopathy, and people without heart disease. Participants enrol and upload their own data through the website. It is the second research study that will be offered to registry participants and delivered through The Heart Hive platform.
A phase 2/3 multi-center randomized, double-blind, parallel arm, placebo- controlled study in Adult Subjects Hospitalized with Severe SARS-CoV-2 Positive Pneumonia to determine the potential of opaganib to improve and/or stabilize the clinical status of the patient.
The study aims to examine the effects of a sleep extension intervention on the metabolic and cardio-vascular profile of obese people who present traditional diabetes risk factors, and who are habitually sleep deprived. Participants randomized to the intervention arm will complete a 6-week sleep extension intervention, whilst the control group will maintain their habitual sleep schedule. It is hypothesized that the sleep extension intervention will significantly increase total sleep time, and will be accompanied by significant metabolic-related changes.
People with lung conditions can suffer significantly with their symptoms and often require multiple trips to their GP or secondary care before a diagnosis is made. It can be difficult to diagnose lung disease and to differentiate between different lung conditions, so many people may be misdiagnosed or incorrectly not given a diagnosis. The tests currently used to diagnose lung diseases can be difficult or uncomfortable to do, especially if the person is experiencing lots of symptoms, and therefore can give poor or unreliable results.A new quick and easy to perform test is needed that can differentiate between various lung conditions and people with no lung disease, that can be performed in primary and secondary care with immediate results.
This study aims to evaluate several of Owlstone Medical's Breath Biopsy aerosol respiratory droplet capture techniques for the detection of nCOVID-19. These are single use disposable breath capture devices with removable filters and fitted PVA strip that directly sample exhaled breath aerosols and therefore, directly sample the primary transmission route for the virus. They can be used independently and shipped for analysis for the presence of nCOVID-19 using established existing assays available in any reference lab. The trial is a non-inferiority trial comparing diagnostic accuracy of collection via face mask vs. available diagnostic procedures in standard care and will also asses the feasibility of patient use of the equipment. Subjects will be recruited at the Addenbrookes Hospital, Cambridge. Minimal patient characteristics (e.g. age, sex) are collected. Between 20 and 100 subjects with with a positive nCOVID-19 diagnosis will be sampled to obtain to address the primary study hypothesis. Due to the pre-test probability of patients being positive for nCOVID-19 is unknown we will monitor the number of sampled subjects with a positive diagnosis on a weekly basis. The clinical diagnosis of the subject based on a combination of imaging, viral diagnostics and clinical assessment will be used as the reference standard. When this number hits 100 the study will be discontinued. In total no more than 500 subjects shall be sampled in this trial.
This study is an observational registry of children with or suspected to have SARS CoV2 (COVID-19) admitted to pediatric intensive care units (PICU). This registry will help describe the prevalence, rate and severity of acute kidney injury (AKI) in children with Severe Acute Respiratory Syndrome Coronavirus-2(SARS CoV2) across the world. The registry will be developed using a point prevalence methodology and then full retrospective review. Once a week, from April through June 2020, data collection will occur in "real-time" to estimate a weekly point prevalence of AKI and renal replacement therapy (RRT). The operational definition of "patients under investigation" (PUIs) will be used to identify the denominator of patients to be studied. The PUIs will be cohorted into SARS CoV2 test positive, test negative, test pending, or test unavailable. The primary aim of this study is to deliver a global, objective data driven analysis of the burden of AKI in virus positive patients or patients under investigation (PUI) who are admitted to the pediatric intensive care unit.
Semi-structured qualitative interviews will be conducted to understand key factors that would enable / facilitate patients with chronic breathlessness to potentially use an online breathlessness intervention (SELF-BREATHE).