This is a prospective, observational, non-interventional, multi-center, global clinical study of subjects with early to advanced stage STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene, confirmed genotypically by an accredited genotyping laboratory. The study will consist of 6 visits over a 96-week study period: Visit 1 (Screening Visit), Visit 2 (Week 16), Visit 3 (Week 32) Visit 4 (Week 48), Visit 5 (Week 72), and Visit 6 (Week 96). Subjects who discontinue the study early will ...

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Atrial Fibrillation (AF) is the most common cardiac arrhythmia and carries an increased risk of stroke. Stroke risk is commonly assessed using the CHA2DS2-VASC scoring system. To reduce stroke risk, guidelines advise AF patients with higher CHA2DS2-VASC scores be treated with oral anticoagulation (OAC), also known as blood thinning medication. OAC reduces the stroke risk, however it also increases the risk of bleeding episodes, which can be major and life-threatening. AF patterns can vary between patients. One patient may have only one ...

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Traumatic Brain Injury (TBI) is caused by a trauma to the head from a force outside the body, like an accident or fall. Around one million people visit Accident and Emergency each year following a head injury. Although many of these people will experience no lasting effects, others will be left with varying degrees of damage that can have devastating and lifelong consequences. Moderate head injury is defined as loss of consciousness between 15 minutes and six hours, or a ...

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Broken ankles in children often involve the area from which the bone grows – the growth plate. Following growth plate injuries, the growth of the main shin bone in the lower leg (the tibia) can be altered permanently, which can cause the bone to not grow at all, or to grow wonky. The younger the child at the time of injury (i.e. the more they have to grow) the worse the problem may get once the child has fully grown. ...

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Survivors of childhood cancer experience impaired health-related quality of life, psychological distress, and pain. A previously developed body mindset intervention has improved health-related quality of life and symptom distress in adult cancer patients. In the current research, we will examine whether a body mindset intervention can instil adaptive body mindsets, boost resilience, and improve physical and psychological functioning in survivors of childhood cancer. Survivors of childhood cancer will be randomised to the body mindset intervention or an active attention control group. ...

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Perianal fistula in Crohn's Disease (CD-pAF) is an abnormal tunnel that develops between the end of the bowel and the skin near the anus. It affects one third of Crohn's Disease (CD) patients causing significant morbidity and poor quality of life. Patients with CD-pAF are treated with several combined approaches involving medical and various surgical techniques with high failure rates (around 50%). The study will provide an evidence base to identify predictors of fistula healing, potentially modifiable factors, ...

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Crohn’s disease is a lifelong inflammatory illness that causes people to have severe stomach pains, chronic diarrhoea, and suffer weight loss. There is no cure. Crohn’s disease causes inflammation, ulceration, bleeding and narrowing of the digestive system. People can have periods of good health (“remission”) and times when symptoms are more active (“flare ups” or “relapses”). Medications can help keep it in remission, however one third of people will need surgery to remove/repair part of their diseased gut at some ...

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Controlling persistent pain is a significant unmet need for people living with Inflammatory arthritis (IA). There is emerging evidence that patients with IA exhibit different types of pain and central sensitisation contributes significantly to chronic pain. The treatment for different pain types will differ. One way in which we might rapidly improve the treatment of pain in IA is by identifying an individual’s pain type in the clinic, then tailor their pain treatment accordingly. Currently this is not routine practice and ...

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Oesophageal squamous cell cancer (OSCC) is a cancer that grows in the food pipe and affects up to 2,000 people in the UK every year. Most patients are diagnosed when the cancer has spread to other body parts (advanced stage). As a result, the five-year survival is less than 20%. In comparison, when diagnosed early, nearly three out of four patients survive more than five years, indicating that early diagnosis improves survival. There are numerous challenges in detecting OSCC early. ...

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The purpose of this study is to evaluate the safety and human clinical response to SARS-CoV-2 challenge with the omicron variant virus, in previously SARS-CoV-2 vaccinated people. We aim to establish the optimal challenge dose that causes re-infection and identify the lowest level of infectious dose necessary to produce viral replication in the upper respiratory tract of research volunteers while minimising risk of disease progression. Investigators aim to achieve an infection model which results in no symptoms, or symptoms no ...

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