Three main arrhythmia syndrome phenotypes currently included in the Genomics England (GEL) 100K Genomes Project are: long QT syndrome, Brugada syndrome, and catecholaminergic polymorphic ventricular tachycardia (CPVT). These disorders are responsible for much of unexplained sudden cardiac death (SCD), an important cause of death in Western countries. A substantial proportion of arrhythmia syndromes are presumed to be due to rare and common genetic variation, however their genetics are variably understood at present and no condition has been ascertained completely. We ...

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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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Myeloma is a treatable, but incurable, malignancy of plasma cells in the bone marrow (BM). It represents the final stage in a continuum of plasma cell disorders (PCDs) and is consistently preceded by a premalignant phase termed monoclonal gammopathy of undetermined significance (MGUS). MGUS therefore provides the opportunity to study how a premalignant condition progresses into cancer. MGUS-to-myeloma progression requires multiple genomic events and establishment of a permissive bone marrow microenvironment (BME). Historically, obtaining patient material to study MGUS has ...

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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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AIM To explore if it is possible to carry out a study testing a fatigue self-management programme for people who survive an intensive care unit (ICU) admission. WHAT DO WE KNOW? Patients with life-threatening illness require care in an ICU. Approximately half of those who survive (approx. 82,000 per year) report fatigue lasting for a year or more. Fatigue is described as an overwhelming physical and mental exhaustion, not relieved by rest or sleep. Fatigue has a devastating impact on ...

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Type 1 diabetes (T1D) is a chronic disease, affecting around 1 in 350 children under the age of 15. Treatment is with life-long insulin and is typically started as an emergency when children present with dangerously high glucose (sugar) levels to their doctor. However, T1D has recognised stages before symptoms develop, providing an opportunity for early diagnosis, education and treatment before severe illness. Tests which help diagnose patients and tell doctors when a person may be soon need insulin ...

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This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, global clinical study to assess the efficacy, safety, tolerability and long-term clinical outcomes of pegozafermin administered once-weekly (QW) subcutaneously (SC), in approximately 762 subjects with compensated cirrhosis due to MASH (fibrosis stage F4 per NASH CRN criteria). The study schema is shown in Section 1.2. The study will enroll a broad spectrum of subjects with compensated cirrhosis, comprising those with or without CSPH. The study will enroll approximately 532 subjects with low ...

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