This modular, multi-part, multi-arm, Phase 1/2, FIH study allows the evaluation of the safety and tolerability of CT7439, dosed as a monotherapy and in combination with anticancer treatment in participants with solid malignancies. Module 1 will initially focus on monotherapy in participants with locally advanced or metastatic solid malignancies and include dose escalation cohorts (Part A) and dose optimization cohorts (Part B) (Appendix A). In Parts A and B of Module 1, a SRC, consisting of study investigators and sponsor medical ...

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Subjects will enter this study after participating in a GTX-102 clinical trial. All subjects will undergo assessments at Screening/Month 0 and throughout the study as indicated in Table 3. Subjects will receive treatment with GTX-102 via IT LP. Upon study entry, subjects will maintain the same dose frequency as they received in the prior GTX-102 trial – either Q2M or Q3M (Figure 1). For the purposes of this study, a month is defined as 28 days. Clinical outcome assessments (eg, Vineland Adaptive ...

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Acute myeloid leukaemia (AML), is an ‘aggressive’ blood cancer with only around 20% of affected adults surviving at 5 years. AML often presents as a medical emergency and treatment is intensive. After the initial course of urgent anticancer therapy, people with AML receive repeated intensive courses of chemotherapy aimed at cure, that may include ‘haemopoietic’ (blood) stem cell transplantation. They need to remain in hospital during courses of treatment, often in isolation, due to risk of infection, with short periods ...

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1. Study design 1.1. Patient and Public Involvement (PPI) PPI remains central to this project,with the Anthony Nolan Patient Services team co-developing the proposal alongside a Patient Advisory Group (PAG). The PAG,consisting of 6 allogeneic HCT patient representatives,has been expanded to include members from underserved and minority groups to ensure diverse perspectives. PAG members actively participated in online meetings and in-person workshops,contributing to research design and sampling timepoints,providing feedback on questionnaire content and measures,and assisting in the development of patient-facing study ...

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The EXPERTS-ALS platform is designed as a randomised,open-label,multi-arm trial with a biomarker-based endpoint. It aims to prioritise drugs for further testing based on their ability to lower NFL levels and to also identify drug-specific adverse events.The EXPERTS-ALS protocol describes an overarching trial design to evaluate the effect of candidate drugs on blood NFL levels in patients with ALS receiving usual standard of care. The protocol is deliberately flexible,allowing: as broad a range of ALS patients to be recruited; participant randomisation ...

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This study will evaluate long-term safety and efficacy in approximately 400 subjects who were dosed with SRP-9001 in a previous clinical study. The study will consist of 2 periods as follows: • An Eligibility Confirmation Visit of up to 2 days in length, during which eligibility confirmation, informed consent, disease characteristics, and current therapy/ concomitant medication data will be collected. • A Long-Term Follow-Up Period that will begin the day eligibility is confirmed and continue for at least 5 years post-infusion of ...

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The purpose of this study is to investigate if it is worthwhile prescribing medicines to reduce blood clots after varicose vein procedures. People enrolled in the study will undergo an assessment to make sure that they don’t have the most important risk factors for clots. Those most at risk would not be included in the trial. People enrolled in the study will be over 18 years of age and would receive compression stockings along with one of the following three ...

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BACKGROUND Venous thromboembolism (VTE) is a major global health burden. Many people who survive VTE suffer from longer term physical complications or psychological sequelae. Temporary lower limb immobilisation (plaster cast,walking boot or rigid splint) and injury are combined risk factors for VTE. Approximately 70,000 people are immobilised after injury every year in the UK. VTE in this setting is potentially preventable through early prophylaxis with anticoagulant medication. However,baseline VTE risk varies widely across the population and there are multiple drugs for ...

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This is a multicenter RWE study to evaluate the efficacy of etrasimod daily dose of 2 mg in patients with moderately to severely active UC. The study consists of a 52-Week treatment period, and a 28-day safety follow-up period. This prospective, international, non-interventional primary data collection study will be conducted in countries across the world. Countries will be divided into study regions. A country can be a study region on its own. Each study region will start with patient recruitment as soon ...

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Overview and Purpose: Preterm birth (PTB) is defined as the occurrence of live birth before 37 weeks of pregnancy. Although PTB affects 10% of pregnancies worldwide and is a major global health concern, its aetiology is not fully understood. Recent studies indicate specific genetic factors are related to PTB. This nationwide multicentre cohort study aims to explore the genetic factors linked to spontaneous preterm birth (sPTB) using Whole Genome Sequencing (WGS) across diverse populations in England. The study may help ...

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