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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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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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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This is a randomized, double-blind, placebo-controlled, parallel-group, multicenter trial in which the efficacy, safety, and pharmacokinetics of orally administered buloxibutid, either as monotherapy or on top of stable IPF therapy, are assessed in participants with IPF. The trial will include participants who are on stable licensed IPF therapy or who are currently not treated with a licensed IPF therapy. The latter group will include participants intolerant or not responsive to licensed IPF therapies, participants ineligible to receive these therapies and participants ...

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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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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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Mitochondria are crucial components found in most cells that generate energy from food to enable cells to function normally. “Spelling mistakes” (mutations) in the POLG gene, cause mitochondrial dysfunction and subsequently disease, in organs with high energy demands. Cerebellum, the part of the brain that regulates our balance and movement coordination, and peripheral nerves, are frequently affected in people with POLG-related mitochondrial disease. They develop ataxia, which is a medical term describing symptoms related to balance impairment and muscle incoordination, ...

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