All Clinical Trials in United Kingdom

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001). Read Less

A cross-sectional retrospective study of a sample of 20 women who completed single agent or multi agent chemotherapy: between 6 weeks and 24 months post treatment involving a semi structured telephone interview. A patient sample of 20 is proposed for the study. These are all the patients who meet the inclusion criteria below and are thus eligible for the study. These patients will be contacted via telephone by the principal investigator to inform them of the study and invite participation. A proposed sample size of 20 is sufficient to generate data to address the central questions and furthermore, this sample size is adequate because the intention is to gain insight into the experiences of patients' perceptions about their psychological experiences. Objectives: * Gaining insight into the emotional impact of GTN post treatment * Ascertaining if health professionals are providing adequate psychological support * Identifying sources of support that patients accessed post completion of treatment * Identifying potential areas of improvement in the follow up support for future patients Criteria for inclusion: * Treated with chemotherapy for a GTN diagnosis * Completed treatment between 6 weeks and 24 months * Are able to provide informed consent * Have no cognitive impairment as judged by the treating clinician Criteria for exclusion * Treatment received less than 6 weeks ago * Treatment received more than 24 months ago * Non-English speaking Outcome measures are not appropriate in this qualitative study. However outputs from this study include increasing knowledge and insight into: * patients' experiences of their psychological experiences post chemotherapy * patients' perspective of the support received after their treatment * potential areas of improvements in care Read Less

This is a Phase 2, randomized, double-blind, placebo-controlled, adaptive, multicenter study to evaluate the efficacy, safety, tolerability, Pharmacodynamics (PD), and Pharmacokinetics (PK) of OATD-01 in the treatment of subjects with active pulmonary sarcoidosis. Read Less

The goal of this prospective, cross-sectional study is to test the strength of the soleus muscle in participants who have a ACL tear confirmed by MRI. Previous studies have shown that the soleus muscle, which one of the muscles situated in the calf, decreases mechanical loading on the ACL. Therefore, we aim to test soleus muscle strength using a seated plantar flexor test. The main questions we aim to answer are: * Do patients with ACL tears demonstrate inter-limb soleus strength asymmetry (\>10%) * Do patients with ACL tears have soleus muscle weakness compared with uninjured (normative) participants? * Do changes in soleus strength values correlate with patient reported outcomes and stability, as measured by the IKDC questionnaire? To achieve this the VALD force frame will be used as the testing device for plantar flexor strength testing. This device records the force generated by the movement. This will be repeated three times on each leg as part of the testing procedure. Furthermore, as part of the testing procedure participants will be required to fill out an IKDC form to assess their knee stability. Interrater reliability of the force frame will be investigated. Further data from participants without ACL tears will be used to compare results. Thus allowing us to see if people with ACL tears have a weakness in the soleus muscle. Read Less

Non-invasive ventilation (NIV) is commonly offered to people with Motor Neurone Disease (MND) who have breathing difficulties. It improves their quality of life and can prolong life by 6 months or more. It is initially used at night and typically set up during a hospital admission. By the time that they develop respiratory failure and need to start NIV, however, most patients require wheelchairs or have other significant health problems. Repeated travel to hospitals is increasingly difficult with increasing disability. It is possible to start and monitor NIV treatment at home. This may be more convenient for selected patients, though starting NIV is quite complex; it is not known if home treatment is as safe and effective as hospital-based treatment. To establish this, 60 patients with MND who have indications for NIV will be recruited. They will be randomly allocated to a home-based treatment (home NIV set up plus home visits supported by telemonitoring) or hospital-based care (inpatient NIV set up plus outpatient NIV monitoring) and followed up at 1, 4 and 7 months. Alongside measures of treatment effectiveness, assessment of patient and carer preferences, quality of life, and cost-effectiveness will be undertaken. In the additional qualitative part on this study, interviews with patients who took part in the main study and their carers will be conducted to understand in more depth their perspective on what makes for a good or bad experience with NIV, how the environment (home vs hospital) influences their NIV experience and what personal factors determine NIV use. Findings from the interviews will inform the design of a truly patient-centred NIV service. Read Less

This research aims to explore differences in walking and functional movement mechanics, exercise response, and physical activity between people with osteoarthritis (OA), those who have undergone total joint replacement, and healthy individuals. Read Less

The aim of this study is to identify the optimal dose for Vosilasarm (EP0062) as monotherapy and in combination with standard-of-care therapies to assess its Safety, Tolerability, Pharmacokinetics, and Efficacy in Patients with Relapsed Locally Advanced or Metastatic AR+/HER-2-/ER+ Breast Cancer Read Less

The goal of this study is to investigate how a common antidepressant citalopram (which increases the levels of the chemical messenger serotonin), affects how a key area of the brain involved in depression (the amygdala) responds to emotional information. Healthy participants will undergo medical and psychiatric health screening, after which they will be assigned to receive either a single dose of citalopram (20mg) or placebo, and undergo brain scanning (7T fMRI) whilst viewing emotional faces. Since the scan uses high field strength, the investigators will be able to see effects of citalopram on different subfields within the amygdala which will help to understand how citalopram might be working. Read Less

This study aims to test if the VR games could be a form of upper limb rehabilitation for people with arm/hand problems due to SCI while they are in hospital. Participants who have had a spinal cord injury and have tetraplegia will be recruited from the Queen Elizabeth National Spinal Injuries Unit. After they have provided informed consent, they will receive 12 weeks of either standard upper limb therapy ('control group'), or both the VR activities and standard treatment ('VR Group'). Participants who receive VR activities will engage in immersive VR games that have been designed in co-production with people with tetraplegia and spinal cord injury specialists. The games aim to help participants improve the use of their arms and hands while they are undergoing primary rehabilitation within the Queen Elizabeth National Spinal Injuries Unit (QENSIU). The participants who are in the control group will receive their usual rehabilitation and will be given the opportunity to try the VR games after the completion of their involvement in the trial. This study will measure the feasibility (the 'primary outcome') and explore the effectiveness (the 'secondary outcome') of the VR intervention. Feasibility will be measured by recording how often the VR games are used and whether or not participants use the games for the full duration of the trial. Participants and therapists will be interviewed at the end of the trial. Read Less

The goal of this study is to determine the efficacy of the study drug olutasidenib to treat newly diagnosed pediatric and young adult patients with a high-grade glioma (HGG) harboring an IDH1 mutation. The main question the study aims to answer is whether the combination of olutasidenib and temozolomide (TMZ) can prolong the life of patients diagnosed with an IDH-mutant HGG. Read Less

This study aims to explore an alternative method for assessing a participant's readiness for surgery, specifically for those undergoing surgery for gastric, oesophageal, or pancreatic cancer. Surgery for these diseases is extremely demanding on the body and surgeons need to be confident that a patient can tolerate the procedure. Traditionally, participants undergo Cardiopulmonary Exercise Tests (CPET) to determine their fitness for surgery. This involves cycling on an exercise bike with increasing resistance until the resistance is too great to continue cycling at a constant rate. An individual's maximum rate of oxygen consumption (VO2max) and the anaerobic threshold (AT) are calculated during CPET and used by surgeons as a predictor for surgical fitness. CPET tests can be physically demanding for participants and are very resource intensive. Furthermore, some patients may not be able to tolerate exercise tests due to physical constraints. An alternative method, Magnetic Resonance Spectroscopy (MRS), could be used to measure an individual's fitness prior to surgery. Within this study, MRS will be used to determine the levels of fat within the thigh muscle, which in turn may be able to be used to predict fitness for surgery. The first phase includes recruiting participants on the surgical pathway. MRS will be performed as an additional examination, typically on the same day as the CPET. Fat levels determined from MRS will be compared with conventional CPET outcomes (VO2max and AT). This analysis aims to determine if MRS derived fat levels correlates with VO2max and AT and, if correlation is high, potentially providing an alternative to CPET for patients who cannot tolerate exercise tests. The second phase involves recruiting volunteers who will undergo repeated MRS scans to assess the reliability of MRS methods. This process allows us to understand the consistency of MRS derived fat levels over both short and long-term intervals. The potential benefits of this project include an additional pathway for assessing fitness for surgery or enhancing the presurgical pathway for patients by providing an alternative method for fitness assessment. This study aims to provide evidence that could present an alternative method of assessing a patient's fitness for surgery. The goal is to explore an alternative that may complement or refine current practices in preoperative assessments. Read Less

The study will evaluate the efficacy and safety of transcatheter aortic valve implantation (TAVI) using the Hydra transcatheter aortic heart valve (THV) series, in patients with severe aortic stenosis up to 1-year after the procedure. This will include an evaluation of the preservation of coronary access post implant by attempting to selectively engage both the right and left coronary arteries through standardised angiographic views. The study will also examine the utility of 72 hours of post discharge remote ambulatory continuous electrocardiogram (ECG) monitoring using the novel Checkpoint Cardio System where it is clinically appropriate. Read Less