We are testing a drug called ginisortamab that blocks a protein called gremlin-1. Gremlin-1 is mainly found outside cancer cells and stops the function of other proteins called bone morphogenetic proteins (BMPs). BMP proteins work by suppressing cancer cells as they occur but in cancer, this mechanism has often been switched off. Blocking gremlin-1 with ginisortamab will allow BMP protein function, and we hope this will change the way the cancer cells develop, making them more sensitive to chemotherapy. We are testing ginisortamab ...

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Research question In patients who have a high-risk primary cutaneous squamous cell carcinoma (HR-cSCC) surgically excised with clear margins,is adjuvant radiotherapy plus close clinical follow-up superior in reducing the risk of loco-regional recurrence compared with close clinical follow-up alone,and if so,is it cost-effective? Background cSCC is the second most common skin cancer in the UK,with annual incidence exceeding 52,000,increasing by 6% p.a (1,2). Treatment,usually surgery,is curative in 95% (3,4). However,5% of patients develop loco-regional recurrence (LRR) which causes 75% of cSCCspecific mortality,substantial morbidity and reduced quality of life (QoL) ...

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The proposed trial tests whether the addition of revumenib,compared to placebo,to azacitidine/venetoclax improves outcome in newly diagnosed adult patients with NPM1-mutated AML,who are not considered candidates for intensive chemotherapy by measuring overall survival. Newly diagnosed,previously untreated patients ≥18 years of age with NPM1-mutated or KMT2A-rearranged AML that are eligible for the study will be randomised in a 1:1 ratio to therapy with azacitidine/venetoclax with revumenib or placebo. Treatment will be on a continuous 28-day cycle schedule and continued until disease progression,development ...

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The proposed trial test whether the addition of venetoclax,compared to placebo,with azacitidine and ivosidenib improves outcomes in newly diagnosed adult patients with IDH1 mutated AML,who are not considered candidates for intensive chemotherapy by measuring event free survival.. Newly diagnosed,previously untreated patients ≥18 years of age with IDH1-mutated or IDH1 mutated MDS/AML that are eligible for the study will be randomised in a 1:1 ratio to therapy with ivosidenib /azacitidine with venetoclax or placebo. Treatment will be on a continuous 28-day cycle ...

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A break to the bone of the upper arm at the shoulder is a painful injury. It results in a sudden loss of use of the arm with recovery taking many months. Most injuries occur in people over 50 years of age after a fall, due to reduced bone strength, and are usually treated with a sling, although some fractures may need surgery. Currently, people are asked to see a physiotherapist a number of times to help with recovery after ...

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The PRESERVE trial aims to show that using our MRI reporting system we can substantially reduce the number of patients having major surgery for early rectal cancers (ERC). Bowel cancer is the UK’s third most common cancer. Major surgery removing a large section of bowel and surrounding tissue is routinely recommended as treatment for rectal cancer as per current guidelines. 1 in 5 patients require a permanent colostomy. It is widely reported by patients that their quality of life (QOL) ...

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Background Paediatric Rheumatology is a term that covers over 80 conditions, affecting different parts of the body, such as Juvenile Arthritis. Children and young people (CYP) with rheumatological conditions can have high levels of mental health problems and therefore are at risk of poor health outcomes. We need to look at novel ways of providing early, essential support, to improve their current wellbeing. Our aims This study will create a digital-service that can guide parents through ways of supporting their CYP at ...

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The study plans to invite individuals with Myasthenia Gravis (MG) from several clinics in England, as well as healthy controls without MG, to visit a research facility. Participants will be asked to provide video and audio recordings using a smartphone device while they complete four standardized assessments. These assessments aim to identify speech problems (dysarthria and dysphonia), arm fatigue/weakness, and droopy eyelids (ptosis). Participants with MG will also complete several standard questionnaire-based MG assessments. The research team aims to investigate the ...

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This study is a multi-centre observational, prospective registry of patients with high-risk neuroblastoma treated with Dinutuximab beta. Pain is an almost universal toxicity associated with anti-GD2 immunotherapy, and can be severe despite opioid analgesia. The aim of the study is to better understand the mechanism of pain is patients receving this immunotherapy, with the ultimate aim of designing better, less toxic anti-GD2 antibodies. The study is non-interventional. All treatment decisions are made according to normal clinical practice and are not ...

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Inflammatory arthritis usually prevents people from doing things through causing joints to become swollen and painful, or their spine to become stiff. People with arthritis usually require long-term treatment with medications and are typically reviewed in outpatient clinics every 6-12 months. Many of these appointments are unnecessary as patients are well at the time of follow-up, wasting patients’ time and NHS resources. NHS England has proposed that many people with inflammatory arthritis should no longer have routine follow-up appointments, but be ...

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