Clinical trials

Parkinson's can cause a wide range of non-motor symptoms (NMS), including pain and problems with mental health, memory and sleep. These affect the quality of life of people with Parkinson's (PwP) and their care partners (CP). If these issues are not recognised and managed quickly, they can result in escalating problems. Many PwP are unsure of the extent and variety of the NMS and how simple adjustments at home could improve them. We have developed a digital system to help PwP monitor their non-motor symptoms and develop skills to self-manage them. Such a tool needs to be simple to use, safe and effective. We will ask 30 PwP and CPs to use the digital tool for 6 months, and we will monitor how they use the tool. PwP and CPs will be asked if they feel more knowledgeable and confident to manage their own symptoms whilst being better able to discuss a problem with their healthcare professional. A smaller group of participants will discuss their experiences in more detail to help pinpoint aspects that work well and those needing adjustment and development. It is thought that the use of this system will result in improved quality of life and increased knowledge and confidence for managing symptoms while safely reducing the time spent by healthcare professionals on manageable non-motor symptoms.

Transient Ischaemic Attack (also known as TIA or 'mini-stroke') affects about 46,000 people in the UK each year. It is assumed that people recover fully within 24 hours. However, subtle problems with speaking, and confidence with their communication skills can be long-term. This project will be the first in depth exploration of speaking abilities after recent TIA where there seemed to be a full recovery. The researchers will look for 90 volunteers for detailed testing. Thirty people who have had a TIA for the first time. For comparisons, the researchers will also include 60 volunteers without TIA (30 treated to prevent TIA and stroke; 30 who do not receive prevention treatments). Aims and methods: 1. To find out if TIA makes speaking difficult. People will complete speaking tasks in a quiet place. For example, people will be asked to tell us about their weekend, what they think about climate change. 2. To find out if people are concerned about their speech and other thinking skills after TIA. People will fill in questionnaires to help us look into these issues. 3. To find out if speaking abilities and people's own views of their communication change over time (about three months after the TIA). Discovering new knowledge about spoken communication after a TIA diagnosis could change the course of TIA research and care across health professions (speech-language therapy, psychology, audiology, neurology). Future studies could use speaking tasks to scrutinise further the complexity of subtle communication problems after TIA and determine which individuals are likely to have these problems. The project will raise understanding of these issues, enabling affected individuals to seek professional support. Finally, it will also guide development of new TIA recommendations and treatments for these problems thus improving people's quality of life.

* Tinnitus affects one in seven adults long-term * Once present persistently for 4 weeks, tinnitus does not usually disappear * People generally become less aware of, and less affected by, their tinnitus over time * Only one in six people with tinnitus suffers a long-term negative impact on their life * Current treatments can help to reduce the impact of tinnitus, but not silence the sound * Treatments have only so far been tested once tinnitus has been present longer than 6 months * In this study, the researchers are testing whether combining two interventions, in the early stages of tinnitus, is more effective in quieting tinnitus than using one treatment alone in the later stages of tinnitus * One intervention (acoustic ripples) involves playing sounds through headphones for up to 60 minutes per day, and is mostly done in your own time * The other intervention (transcranial direct current stimulation: tDCS) involves applying a weak electrical current to the volunteer's scalp using sponges soaked in salt water. This is performed in Newcastle University Medical School for a total of 8 sessions of 40 minutes each, spread over 4 weeks * Half the volunteers will receive the 'real' intervention, and half a 'sham' or 'placebo' version. This will be randomly determined, and the volunteers or the research team will not known which you are receiving until the end of the study * When the study ends, all volunteers will be able to use the 'real' intervention sounds in their own time, for as long as they wish, if they want to do so. No volunteers will be able to receive tDCS after the end of the study. * All volunteers also complete questionnaires about their tinnitus, related symptoms and general health, and have hearing tests plus EEG (electrical brainwave) recordings * The study is very low-risk, but does involve a total of 10 visits of around an hour each to Newcastle University Medical School over around a month. These can be arranged flexibly, including daytimes, evenings and weekends * The researchers will pay all reasonable travel expenses, and £10 per hour for the volunteer's time in participating * The study only completely ends 6 months after the date the tinnitus first started. At this point, the researchers will send some questionnaires to complete only. There is nothing to do for the study in between the tenth visit and this six-month questionnaire.

Brief summary The aims of this project are to 1) characterise muscle fatigue in individuals with chronic heart failure during exercise involving a smaller and larger muscle mass (Part I), 2) to determine the effect of nitrate supplementation on muscle fatigue during large muscle mass exercise in individuals with chronic heart failure (Part II), 3) understand the impact of exercise intolerance on quality of life in individuals with chronic heart failure (Part III). The main questions it aims to answer are: * Is muscle fatigue attenuated during exercise engaging a smaller vs larger muscle mass in individuals with chronic heart failure owing to lower central cardiopulmonary constraints? * Can supplementation with nitrate-rich beetroot juice reduce muscle fatigue and/or accelerate post-exercise recovery of muscle function in response to whole body exercise in individuals with heart failure? * What impact does exercise intolerance have on the lives of individuals with chronic heart failure? For Part I, researchers will compare muscle fatigue during single- and double-leg incremental cycling in individuals with chronic heart failure. For Part II, researchers will compare muscle fatigue in individuals with chronic heart failure during double-leg incremental cycling following a period of beetroot juice supplementation containing nitrate, or with a placebo drink consisting of beetroot juice with nitrate extracted. For Part III, semi-structured interviews will be conducted to investigate the symptoms associated with performing physical activity and on the impact of exercise intolerance on quality of life in individuals with chronic heart failure.

Heart Failure occurs when the heart's ability to pump blood is reduced. Heart failure can lead to symptoms of breathlessness, fatigue and ankle swelling, and result in health complications including damage to other organs (e.g. kidneys), reduced function and quality of life. Although the symptoms of heart failure are similar for men and women, there are sex differences. Lifestyle behaviours such as physical activity are important modifiable risk factor for heart failure. Women continue to be underrepresented in heart failure studies and treatment guidelines are male-derived due to these disparities in recruitment. The purpose of the present study is to evaluate the physical activity levels, sedentary behaviour, sleep and quality of life and understand the barriers and facilitators to these lifestyle behaviours in women newly diagnosed with heart failure.

Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults. It is an autosomal recessive disorder and is therefore caused by inheritance of a mutated gene from each parent. All forms of SMA have an estimated combined incidence of 1 in 6,000 to 1 in 10,000 live births, with a carrier frequency of 1/40 to 1/60. The patient registry aims to facilitate a questionnaire-based research study in order to better characterise and understand the disease in the UK and in Ireland. Entry is via self-registration over a secure internet connection (https://www.sma-registry.org.uk/). Online, patients are asked to read an information sheet about the research project and then indicate their consent to demonstrate willingness to participate. Following online consent, subjects will be entered into the registry. This is an on-going database and all participants are invited to update their information on a biannual basis.

Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000. FSHD is an autosomal dominant genetic disease and is estimated to affect up to 3,000 people in the UK. The patient registry facilitates a questionnaire based research study to better characterise and understand the disease in the UK, and helps to identify potential participants eligible for clinical trials.

Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene. DM1 is the most common adult-onset muscular dystrophy, and is thought to affect at least 1 in 8,000 people worldwide. The aim is to facilitate a questionnaire based research study in order to better characterise and understand the disease in the UK. By maintaining a national registry this will help identify potential participants eligible for clinical trials in the future.

The European NAFLD Registry is a prospectively recruited, observational study supporting the study of the clinical phenotype, natural history, disease outcomes and pathophysiology of Non-Alcoholic Fatty Liver Disease and Non-Alcoholic Steatohepatitis. The ultimate goals are to better understand the drivers of interpatient variation in disease pathophysiology and severity and to utilise this information to develop and validate biomarkers that, singly or in combination, enable detection and monitoring of disease progression and/or from NAFL through NASH to fibrosis and cirrhosis.