There are about 25435 clinical studies being (or have been) conducted in United Kingdom. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
Severe haemophilia A and B (SHA, SHB) are inherited bleeding disorders affecting male patients and are characterised by low levels of circulating clotting factors VIII and IX respectively. Clinically low levels present with multiple recurrent bleeds into joints and muscle from the first couple of years of life. In addition patients may present with spontaneous and potentially fatal bleeding into any organ. The mainstay of treatment is replacement with the missing factor in the form of intravenous injections of factor VIII and IX. Clotting factors can be given to treat a bleed or can be given to prevent a bleed, and the latter is termed prophylaxis. Regular prophylaxis is the current standard of care and aims to decrease spontaneous bleeding events and resulting joint damage, and this requires patients to self-infuse factor into their veins two to four times week. Patient's compliance with prescribed regimen and recommendations has a significant influence on outcomes. Advances in biomolecular and protein engineering have extended the duration of the effect of clotting factor VIII and IX through multiple mechanisms. This extension of the duration of the effect presents the clinician and patients with opportunities to tailor the treatment to their particular needs, circumstances and body other characteristics. It has been suggested that decreasing the frequency of infusions will improve adherence and thus contribute to improved outcomes. In rare disorders, it is an accepted fact that post-marketing studies are crucial to understand the generalisability of the efficacy and safety outcomes and identify any new safety and efficacy concerns in relation to specific population group. The investigators propose the development of a registry for systematic collection of information with the dual aim of analysing the relationship between patient and treatment characteristics, and outcomes, and simultaneously identify areas for practice development that can improve the overall quality of life experienced by the haemophilia patient community.
Percutaneous Coronary Intervention (PCI) is increasingly effective to treat Chronic Total Occlusion (CTO) lesions in coronary arteries. This trial will examine modern dissection and re-entry approaches to treat more complex CTO lesions with the Absorb Bioresorbable Vascular Scaffold (BVS).
The investigator prospectively compare diagnostic concordance of whole body multi--‐parametric Magnetic Resonance Imaging (MRI) with current conventional multi--‐modality reference standard imaging (CT scan, isotope bone scan +/--‐ PET--‐CT scan) for staging of prostate cancer patients with biochemical relapse following external beam radiotherapy or brachytherapy of locally advanced prostate cancer.
Children and adolescents with Hodgkin lymphoma currently undergo multiple investigations including a separate PET and MRI scans during their treatment. Investigators want to investigate if a combined PET-MRI scan could give the same information. Children who join our study will have an extra scan twice during their treatment
This is a multi-centre, non-randomised, open label Phase I clinical trial of an Advanced Therapy Investigational Medicinal Product (ATIMP) in adults (age ≥16) with (1) high risk, relapsed/refractory (r/r) CD19+ B-ALL; (2) r/r DLBCL; (3) r/r CLL/SLL and (4) r/r FL and (5) r/r MCL. The ATIMP for this study is cryopreserved autologous patient-derived T-cells transduced with the lentiviral pCCL.PGK.alpha.CD19CAT-41BBzeta vector, CD19CAT-41BBζ CAR T-cells (referred to subsequently as CD19CAR T-cells) which is classified as a gene therapy medicinal product. Patients will undergo an unstimulated leucapheresis for the generation of the ATIMP. The ATIMP will take approximately 15 days to generate. During this period, patients may receive "holding" chemotherapy as per institutional practice to maintain disease control. The study will evaluate ATIMP safety and efficacy and the duration of disease response in adults with high risk / relapsed CD19+ B-ALL, DLBCL, B-CLL/SLL, FL and MCL. Recruitment into the ALL cohort has been completed and no further patients with ALL are being treated on the study. Patients receive pre-conditioning lymphodepleting chemotherapy with cyclophosphamide 60mg/kg on Day -6 and fludarabine 30mg/m2 administered over 3 days (Day -5 to Day -3). Patients with DLBCL only will also receive a single dose of pembrolizumab 200 mg at day -1. Patients recruited to ALLCAR19 are treated with different dosing schedules, depending on their underlying disease. Patients with B-ALL and B-CLL/SLL are considered at high risk of CLL/CRES so receive split dosing, with the second dose only given in the absence of severe toxicity 9 days later. CAR T-cell dosing in ALLCAR19 is flat i.e. not dependent on patient body weight or surface area. - Regimen A1: Patients with B-ALL with a baseline marrow blast% of ≤20% receive a split dose with a first dose of 100 x 10^6 CD19 CAR T-cells and a possible second dose of 310 x 106 CAR T-cells - Regimen A2: Patients with B-ALL with a baseline marrow blast% of >20% receive a split dose with a first dose of 10 x 10^6 CD19CAR T-cells and a possible second dose of 400 x 10^6 CAR T-cells - Regimen B: Patients with DLBCL receive a single dose of 200 x 10^6 CAR T-cells - Regimen C: Patients with CLL/SLL will receive a split dose with a first dose of 30 x 106 CD19 CAR T-cells and a possible second dose of 200 x 10^6 CD19 CAR T-cells. - Regimen D: Patients with FL and MCL receive a single dose of 200 x 10^6 CAR T-cells The study evaluates ATIMP feasibility and safety of generating CD19CAR T-cells and for B-ALL patients only, efficacy and the duration of disease response to CD19CAR T-cells. After completing the interventional phase of the study all patients, irrespective of whether they progressed or responded to treatment, enter long term follow up until 10 years post-CD19CAR T-cell infusion.
The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with rVWF for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.
Hepatocellular carcinoma (HCC) is the most frequent primary tumour of the liver and is the third cause of cancer-related mortality worldwide. Depending on the stage of the disease, the treatment options are surgery, liver transplantation, chemotherapy or radiotherapy. Recently, scientific research has focused on small molecules called microRNAs which are produced by human cells and can be released in the blood. They have a role in cell proliferation and are found to be dysregulated in different types of cancer. It has been shown that microRNAs have a role in the development of HCC but it is unknown if these molecules can be used as markers for diagnosis and survival in HCC. In particular, microRNAs miR-221 and miR-222 are dysregulated in the tumoral tissues in about 80% of patients with HCC. This can be assessed on tissues from liver biopsies or surgical specimens, both invasive approaches. Only few studies showed the presence of microRNAs in the blood of patients with HCC but it is unknown if there is a correlation between tumoral tissue expression and circulating levels. The aim of this study is to evaluate if these two microRNAs are expressed not only in the tumoral tissues but also in the blood from cancer patients, and in different amounts compared to circulating levels in healthy individuals. A correlation between tumoral tissue and blood levels will also be evaluated. Should this evaluation show a strong correlation and reliability of circulating microRNAs in the diagnosis and follow up of HCC, future clinical trials targeting these microRNAs and their related pathways might benefit from this being adopted as conventional practice instead of the need of assessing tissue levels from liver biopsies. The results of this pilot study will bring preliminary results as a first step for future analysis on a larger cohort of patients.
The sit-to-stand (STS) movement is a key functional movement critical to independent living. This movement is physically demanding to conduct, especially in older adults, and in the presence of physical impairments associated with a range of conditions, such as stroke, osteoarthritis, Parkinson disease and hip arthroplasty. A limited capacity to perform this movement increases the risk of falls, dependency and increased support for personal care and rehabilitation. Restoring independence in this movement is, therefore, considered a priority for physical rehabilitation. Sit-to-stand capacity can be regained through participating in rehabilitation exercises. Providing feedback on performance of this movement could enhance the training. Thus, it is an essential aspect of physical therapy. Healthcare providers are required to meet the needs of an increasingly frail population as well as meeting national, evidence-based, guidelines for improving outcomes in conditions such as stroke which includes an increase in the practice repetition of functional movements. Reliance on rehabilitation staff to provide practice, however, places a limit on practice volume, potentially restricting outcomes. Using technology to enhance safe, repetitive practice of this movement with minimal supervision from skilled professionals would be beneficial to patients and rehabilitation services. The primary aim of this study is to test the acceptability and feasibility of a STS training system that enhances movement feedback to patients undergoing rehabilitation. A secondary aim is to gather data on the effectiveness of the system compared to conventional rehabilitation. This information will inform a statistically powered phase 2 trial.
This clinical investigation is a medical device trial to examine the safety and efficacy of TheraSorb sFlt-1 adsorber treatment of pregnant patients with preeclampsia.
This study aims to understand the state of onset of NLSD(neutral lipid storage disease) / TGCV(triglyceride deposit cardiovasculopathy) worldwide, background information of affected patients, and natural history of the disease, as well as exploring the prognostic factors and assessing the efficacy of disease-specific treatment.