[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Great Ormond Street Hospital for Children NHS Foundation Trust\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":307},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,11,0,[8,45,69,96,120,157,182,208,229,260,285],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":27,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100624688","advanced-mri-in-ivh-100624688",false,"NCT07412886","Advanced MRI in IVH","Advanced MRI in Preterm Intraventricular Haemorrhage: Uncovering Mechanisms of Disease and Novel Treatments","Neonates with PHVD who have been referred to and\u002For have undergone neurosurgical treatment at Great Ormond Street Hospital (GOSH) and Alder Hey Children's Hospital (AHCH) (15 neonates in each) will be eligible for inclusion.\n\nThe majority of neonates will be recruited to the ENLIVEN-UK randomised clinical trial and involvement will not preclude recruitment to this study.\n\nThe only exclusion criteria will be lack of informed consent from the parent\u002Fcarer.","ALL",{"count":18,"type":19},30,"ESTIMATED","INTERVENTIONAL",[22],"NA","55,000 babies are born prematurely in the UK annually. Bleeding in the fluid spaces of the brain (ventricles) is common after prematurity; in England around 450 babies suffer from severe bleeds every year. This is the most important cause of neurological disability after prematurity. Bleeding occurs in the first week of life when the brain is developing rapidly and is most vulnerable to injury. The blood and its breakdown products in the brain fluid (cerebrospinal fluid, CSF) are toxic to the developing brain and cause scarring that blocks the flow and absorption of CSF. In about half these babies, this causes fluid build-up, or post-haemorrhagic ventricular dilatation (PHVD).\n\nCurrent standard treatment of PHVD only drains CSF to reduce pressure inside the brain. Following early results and a successful pilot study at GOSH, we developed an NIHR-funded randomised national trial to analyse the impact of an operation to wash out blood inside the brain using a small endoscope. We will compare standard treatment (fluid drainage alone) with washout plus drainage of fluid.\n\nPremature babies typically undergo an MRI scan of the brain at their expected birth time to assess their brain injury, predict the severity of their disability and see what early rehabilitation and treatment they need. In this study we will use new MRI techniques during this scan at GOSH and Alder Hey Hospital to better understand the extent of brain injury in relation to brain structure, function and brain fluid flow. We want to see whether these will show the impact of the washout procedure, tell us about how washout works, and improve prediction of the child's disability and early treatment needs. If successful, we will apply for further funding to extend these techniques to the other centres in the UK and maximise their benefit within the NHS.",[25,26],"Intraventricular Hemorrhage Neonatal","Post-hemorrhagic Hydrocephalus",[28,29,30,31],"intraventricular hemorrhage","post-hemorrhagic ventricular dilatation","magnetic resonance imaging","cognitive development","NOT_YET_RECRUITING","2026-02-09",{"date":35,"type":36},"2026-02-17","ACTUAL",{"date":38,"type":19},"2026-02-16",{"date":40,"type":19},"2027-05-01",{"name":42,"class":43},"Great Ormond Street Hospital for Children NHS Foundation Trust","OTHER",2,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":49,"acronym":50,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":20,"phases":55,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":68},"100550951","thalamic-recordings-in-children-undergoing-seeg-100550951","NCT06453759","Thalamic Recordings in Children Undergoing SEEG","TRICS","Inclusion Criteria:\n\n1. All children undergoing SEEG as part of their pre-surgical evaluation at GOSH\n2. Participants\u002Fparents\u002Flegal guardian provide informed consent for inclusion\n\nExclusion Criteria:\n\n1\\) Lack of informed consent","2 Years","20 Years",{"count":18,"type":19},[22],"Stereoelectroencephalography (SEEG) forms a key part of the pre-surgical evaluation in children who may be candidates for epilepsy surgery. It can help delineate the location of the putative epileptogenic zone, guiding further treatments including resective, disconnective and ablative epilepsy surgery techniques. However, less than 35% of children undergoing SEEG end up becoming seizure free following further treatment.\n\nOpen and closed loop stimulation of thalamic nuclei via deep brain stimulation (DBS) and responsive neurostimulation (RNS) are emerging treatment options for epilepsy. Thalamic target nuclei vary between studies and there are currently no gold standard personalised methods for choosing a target. This stems from the limited systematic neurophysiological recordings from thalamic nuclei; investigators currently do not understand the ictal and interictal thalamic signatures of involvement in epilepsy and do not understand how functional connectivity can be altered within and between patients.\n\nIn this prospective study, the investigators aim to recruit 30 patients undergoing SEEG as part of their pre-surgical evaluation for drug resistant epilepsy at Great Ormond Street Hospital over a period of 3 years. Once recruited, the investigators will target 3 nuclei bilaterally in each patient - the anterior, centromedian and pulvinar nuclei - using additional SEEG electrodes. Following clinical recording, the investigators will conduct two stimulation experiments, the first using single pulse electrical stimulation to measure effective connectivity between the thalamus and cortical regions and the second to study the effects of simulated DBS currents on cortical local field potential signatures.\n\nThis study will lay the foundation for a personalised approach to thalamic neuromodulation for drug-resistant epilepsy by identifying neurophysiological biomarkers of thalamic involvement in epilepsy, paving the way for closed loop neuromodulation strategies that aim to optimise response using these biomarkers.",[58],"Focal Epilepsy","RECRUITING","2026-01-29",{"date":62,"type":36},"2026-02-02",{"date":64,"type":36},"2025-09-01",{"date":66,"type":19},"2028-12",{"name":42,"class":43},1,{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":16,"minAge":77,"maxAge":78,"enrollmentInfo":79,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":82,"conditions":83,"keywords":85,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":4},"100601615","calprotectin-and-gut-disease-study-100601615","NCT07112794","Calprotectin and Gut Disease Study","Single-test Accuracy Study to Assess the Diagnostic Value of Faecal Calprotectin to Diagnose Necrotising Enterocolitis in Infants With Heart Defects","SAFE NEC","Inclusion Criteria:\n\n* Infants aged from birth up to 3 months old, of any gestational age who require admission to a specialist cardiac centre for treatment of major congenital heart disease.\n\nIncluded patients are those who are judged as likely to require early cardiac surgery or interventional catheterisation, including babies who are treated with invasive medical therapies of respiratory support or intravenous vasoactive medications and their pathway will include a cardiac intervention of any type.\n\nExclusion Criteria:\n\n* Patients will be excluded if they are older than 3 months, do not have major heart disease and do have any gastrointestinal abnormality, including gastroschisis or imperforate anus.\n\nAlso, patients with neutropenia will be excluded since calprotectin is not activated when there is neutropenia.","0 Months","3 Months",{"count":80,"type":19},300,"OBSERVATIONAL","Necrotising enterocolitis (NEC) is a serious gut disease that can develop in infants born with cardiac defects. It causes inflammation and injury to the gut mucosa and may be life-threatening.1 The aetiology of NEC in cardiac infants is multifactorial, associated with poor cardiac outflow and poorly oxygenated systemic circulation, resulting in suboptimal blood flow to the intestines.2\n\nMaking an accurate and timely diagnosis of NEC is a significant clinical challenge.3 The clinical presentation of NEC is difficult to identify correctly in the early stages of the disease. Of concern, infants who are diagnosed with NEC at a later stage have worse disease.4 Due to the potentially life-threatening effects of NEC, clinicians who suspect a baby has NEC but are unable to confirm it, temporarily manage infants as if they have NEC as a precautionary measure.\n\nThis leads to infants without NEC having their milk feeds stopped for several days and being given unnecessary antibiotics. Compared to infants with confirmed NEC, the care of infants with suspected NEC is widely variable in terms of antibiotic regimen chosen, length of antibiotic treatment, and length of time nil-by-mouth - all these variabilities may adversely contribute to the length of hospital stay.5\n\nWe want to study a method to improve the accuracy of diagnosing NEC. We want to measure a protein marker found in babies' stools that, if combined with currently available tests (clinical features, lab tests and abdominal X-rays), 6,7 may improve the timeliness and accuracy of making a NEC diagnosis, crucially at an early stage of gut disease.\n\nThe protein marker is called calprotectin and monitoring levels in a baby's stools before and after heart surgery may help us to understand its relationship to NEC. Calprotectin is a biomarker that is released by white blood cells and is present in the gut when it is inflamed and can be measured in stool.8 The amount of calprotectin in the stool may indicate the level of gut inflammation and the presence of NEC in infants with heart defects.9\n\nThe addition of calprotectin measurement to the current routine parameters (clinical features, lab tests and abdominal X-rays) may improve the timeliness and accuracy of diagnosing NEC in infants with cardiac defects, hence improving their care pathway and outcomes.",[84],"Congenital Heart Disease",[86,87],"necrotising enterocolitis","Faecal Calprotectin","2025-08-01",{"date":90,"type":36},"2025-08-08",{"date":92,"type":19},"2025-11-01",{"date":94,"type":19},"2027-12-01",{"name":42,"class":43},{"id":97,"slug":98,"hasResults":11,"nctId":99,"briefTitle":100,"officialTitle":101,"acronym":4,"eligibilityCriteria":102,"healthyVolunteers":11,"sex":16,"minAge":103,"maxAge":104,"enrollmentInfo":105,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":107,"conditions":108,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":112,"lastUpdatePostDateStruct":113,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":68},"100585224","gut-health-in-children-with-cancer-100585224","NCT06899581","Gut Health in Children With Cancer","Monitor Gut Health in Children Undergoing Treatment for Acute Myeloid Leukaemia Treatment: Case-control Study","Inclusion Criteria:\n\n* \\- Consented to partake in the study\n* Aged 0-16 years old\n* Diagnosed with AML\u002FHLH\u002F Burkits\n\nExclusion Criteria:\n\n* 17 years old + (treated at UCL adolescent unit)\n* Inflammatory bowel disease: ulcerative colitis or Crohn's disease\n* Children who had previously been treated with chemotherapy in another institution age","1 Month","16 Years",{"count":106,"type":19},40,"The purpose of this study is to observe the impact leukaemia treatment has on gut health (microbiomes) and how quickly the gut health recovers after leukamia treatment. The gut microbiome has a number of important functions not only in the gut but within the whole body. Changes to your child's nutritional status throughout treatment may affect how well they recover from treatment. This study will monitor the impact of feeding and nutrition on nutritional status and gut health in young people undergoing treatment for leukaemia. The measurements needed to observe nutritional and gut microbiomes will occur when your child attends their routine medical appointments at Great Ormond Street Hospital.\n\nMedical treatment uses chemicals to kill leukaemia cells. The type of medications used in the treatment of leukaemia can damage the gut resulting in inflammation call mucositis. This stops the gut from working and sometimes nutrition has to be provided via a feeding tube or intravenous. Chemotherapy, mucositis and intravenous nutrition all have an impact on the gut. Little is know how the gut health recovers after treatment for leukaemia. This will be the first study to specifically monitor the impact of feeding and nutritional on gut health in children undergoing treatment for leukaemia.\n\nBy understanding what changes are occurring to your child's nutritional status and gut halth during treatment and during recovery will help to develop guidelines for healthcare professionals to support optimal gut health recovery.",[109,110,111],"AML (Acute Myelogenous Leukemia","HLH","Burkitt Lymphoma\u002FLeukemia","2025-03-21",{"date":114,"type":36},"2025-03-28",{"date":116,"type":36},"2025-01-30",{"date":118,"type":19},"2026-10-30",{"name":42,"class":43},{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":126,"eligibilityCriteria":127,"healthyVolunteers":11,"sex":16,"minAge":128,"maxAge":129,"enrollmentInfo":130,"targetDuration":4,"studyType":20,"phases":132,"briefSummary":133,"conditions":134,"keywords":139,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":149,"lastUpdatePostDateStruct":150,"startDateStruct":152,"completionDateStruct":154,"leadSponsor":156,"locationsCount":68},"100575288","the-flame-cognitive-rehabilitation-study-for-childhood-brain-tumour-100575288","NCT06770335","The FLaME Cognitive Rehabilitation Study for Childhood Brain Tumour","Strategy-based Cognitive Rehabilitation with Integrated Fatigue Management for Patients with Paediatric Brain Tumour (PBT): an Acceptability and Feasibility Study of the Fatigue, Learning and Memory Enrichment (FLaME) Intervention","FLaME","Inclusion Criteria:\n\n1. Age range: 7 years to 17 years, 11 months.\n2. Received diagnosis and\u002For treatment\u002Fsurveillance at GOSH for a childhood tumour that involved the CNS (brain) and\u002For oncology treatment to brain.\n3. Received or receiving a neuropsychological assessment\u002Fconsultation at GOSH over the course of the study period or in the 48 months prior to the study period, or under active surveillance with the neuro-oncology multidisciplinary team during the study period.\n4. At least 6 months post-diagnosis\u002Facute treatment (surgery and\u002For radiotherapy), and 3 months post-return to school, with stable disease.\n5. One or more scores outside of normal limits (i.e. 1 SD above or below the mean in the direction indicating difficulty) in at least one neuropsychological domain (on performance-based tests or questionnaire-based rating scales).\n6. Report impairment (z-score \\> -0.67) in fatigue on one or more subscales of the PedsQL Multidimensional Fatigue Scale.\n7. Capacity\u002Fcompetence of patient or parent\u002Fcarer to provide informed\n\nExclusion Criteria:\n\n1. Completed or having another targeted formal psychological intervention for cognitive rehabilitation or fatigue in the past 6 months.\n2. Sensorimotor (e.g., visual-motor) impairment only on neuropsychological assessment without additional cognitive difficulty.\n3. Current substance misuse from self-report.\n4. Currently receiving formal psychiatric care for a diagnosed mental health disorder (including active suicidal ideation), excluding ADHD treatment (if a child has a diagnosis of ADHD they should be treated).\n5. Intellectual Disability based on a standard score of more than 2 standard deviations below the mean on a general adaptive behaviour composite and, where available, the General Ability Index of intellect.\n6. Patient and parent\u002Fcarer is unable to communicate verbally and in written form in English.","7 Years","18 Years",{"count":131,"type":19},36,[22],"Medical treatments have improved survival rates for children with brain tumours. However, most children experience long-term difficulties with 'cognition' (thinking skills such as memory and paying attention) and cognitive fatigue (excessive mental tiredness) after treatment. Thinking difficulties and fatigue can affect a child's ability to learn, and their social and emotional wellbeing. National guidance recommends treatment called 'cognitive rehabilitation' which teaches skills to improve or manage cognitive difficulties. Families often request this, but it is not usually available due to little research. Fatigue may also get in the way of children using and benefiting from cognitive rehabilitation. No research study has offered a fatigue treatment for children recovering from brain tumours. The study aims to see if it is practical and helpful to families to provide cognitive rehabilitation for children affected by brain tumours. The treatment focuses on strategies to help cognition. The investigators will see if adding strategies to manage fatigue helps. The study will include thirty-six 7-17-year-olds who have been treated for brain tumour at Great Ormond Street Hospital. All participants will have had an assessment describing cognitive strengths and weaknesses as part of usual care. Participants will be randomly allocated to one of three groups: 1) cognitive rehabilitation with fatigue management (12 weeks), 2) cognitive rehabilitation only (6 weeks), or 3) usual care. Each child and their carer will complete questionnaires before, during, and after the treatment, and an interview at the end of the treatment. This information will help the researchers see if families find the treatment helpful and practical to take part in, and if adding fatigue strategies is beneficial. Researchers will look at information such as the number of appointments attended, feedback about the treatment, and information about fatigue levels, cognition, and wellbeing. The findings will be used to develop a UK-wide study.",[135,136,137,138],"Childhood Brain Tumor","Childhood Brain Tumors","Pediatric Brain Neoplasms","Pediatric Brain Tumor",[140,141,142,143,144,145,146,147,148],"Paediatric brain tumour","cognitive rehabilitation","cognition","fatigue","feasibility","acceptability","neuropsychology","neurocognitive","childhood brain tumour","2025-01-07",{"date":151,"type":36},"2025-01-13",{"date":153,"type":19},"2025-03-10",{"date":155,"type":19},"2026-12-31",{"name":42,"class":43},{"id":158,"slug":159,"hasResults":11,"nctId":160,"briefTitle":161,"officialTitle":161,"acronym":4,"eligibilityCriteria":162,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":129,"enrollmentInfo":163,"targetDuration":52,"studyType":81,"phases":4,"briefSummary":164,"conditions":165,"keywords":170,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":174,"lastUpdatePostDateStruct":175,"startDateStruct":177,"completionDateStruct":179,"leadSponsor":181,"locationsCount":68},"100341147","infant-and-child-european-cryoablation-project-icecap-100341147","NCT03721783","Infant and Child European Cryoablation Project ICECaP","Inclusion Criteria:\n\n* all children in whom a clinical decision is made to treat with percutaneous cryoablation, to include those in whom cryoablation was not technically or clinically possible at any stage.\n\nExclusion Criteria:\n\n* none",{"count":106,"type":19},"A registry to audit the current range of applications, technical success rate, safety profile, complication rate and efficacy of cryoablation procedures being performed in children.",[166,167,168,169],"Therapy","Soft Tissue Lesion","Cryoablation","Pediatric",[171,172,173],"cryoablation","pediatric","registry","2024-10-24",{"date":176,"type":36},"2024-10-26",{"date":178,"type":36},"2021-06-24",{"date":180,"type":19},"2027-07-01",{"name":42,"class":43},{"id":183,"slug":184,"hasResults":11,"nctId":185,"briefTitle":186,"officialTitle":187,"acronym":188,"eligibilityCriteria":189,"healthyVolunteers":11,"sex":16,"minAge":128,"maxAge":190,"enrollmentInfo":191,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":193,"conditions":194,"keywords":196,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":200,"lastUpdatePostDateStruct":201,"startDateStruct":203,"completionDateStruct":205,"leadSponsor":207,"locationsCount":68},"100555961","medium-to-long-term-outcomes-of-selective-dorsal-rhizotomy-100555961","NCT06518889","Medium to Long Term Outcomes of Selective Dorsal Rhizotomy","Medium to Long-term Outcomes After Selective Dorsal Rhizotomy in Ambulatory Children and Young People With Cerebral Palsy: A Mixed-methods Study","MOSAiC","Inclusion Criteria:\n\n* Children and young people with cerebral palsy (CYPwCP)\n* Classified as GMFCS level II and III (ambulatory)\n* Aged between 2-15 years at the time of surgery\n* Underwent SDR surgery at Great Ormond Street Hospital for Children (GOSH) (between 2013- 2021)\n* Had baseline (pre-surgery) assessment and at least one follow-up assessment at 6, 12 or 24 months post-SDR\n\nParents of CYP with CP who have undergone SDR surgery (and meet the above criteria for CYP)\n\nExclusion Criteria:\n\n* Children with CP classified as GMFCS level IV, V (non-ambulatory)\n* Have any unrelated musculoskeletal problems, such as a recent acute injury","90 Years",{"count":192,"type":19},90,"The main purpose of this study is to investigate the medium to long-term outcomes (3-10 years) after Selective Dorsal Rhizotomy (SDR) in ambulatory children and young people with cerebral palsy.\n\nThe participants will complete a survey, come to hospital for some measurements and tests.\n\nSome parents and children and young people will also be invited to take part in an interview to understand their experiences of SDR.",[195],"Cerebral Palsy",[197,198,199],"Selective Dorsal Rhizotomy","Rehabilitation","Physiotherapy","2024-07-18",{"date":202,"type":36},"2024-07-24",{"date":204,"type":36},"2024-06-25",{"date":206,"type":19},"2026-05",{"name":42,"class":43},{"id":209,"slug":210,"hasResults":11,"nctId":211,"briefTitle":212,"officialTitle":213,"acronym":4,"eligibilityCriteria":214,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":215,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":217,"conditions":218,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":204,"lastUpdatePostDateStruct":222,"startDateStruct":224,"completionDateStruct":226,"leadSponsor":228,"locationsCount":4},"100553087","seen-and-be-heard-ensuring-fair-cancer-care-for-all-children-100553087","NCT06481527","Seen and Be Heard: Ensuring Fair Cancer Care for All Children","The Seen and Be Heard Study: Identifying the Barriers and Facilitators to Ensuring Equitable Cancer Care for Children With and Without a Learning Disability and\u002For Autism Spectrum Condition.","Work package 1\n\nA) National staff survey\n\nInclusion criteria:\n\n* Healthcare professionals involved in cancer care delivery\n* UK based\n\nExclusion criteria:\n\nOutside stated of location\n\nB) Organisational survey\n\nInclusion criteria:\n\n\\- Clinical service leads\n\nC) Anonymised retrospective electronic case note review\n\nInclusion criteria:\n\nChildren and Young aged 0-16 with Cancer diagnosis between April 2017 - present, within one large children's hospital site.\n\nWork Package 2 - Ethnography study (including interviews)\n\nInclusion criteria\n\n* Children and Young people receiving cancer care, with\u002Fwithout learning disability and or who are autistic at hospital research site\n* Ages 5-15\n\nExclusion criteria\n\n* Within palliative care at the time of recruitment\n* Subject to child safeguarding proceedings\n* Outside of stated age range\n* Children who have not been disclosed their cancer diagnosis and where their family would prefer to maintain a complete non-disclosure approach\n\nParents:\n\nInclusion\n\n* Of children receiving cancer care in hospital research site\n* If included for interview only, child's age may be aged 0-16\n* Conversational English\n\nExclusion - Has mental health difficulties that would impair ability to participate effectively or create an unmanageable risk e.g. psychosis\n\nHealthcare professionals:\n\nInclusion\n\n\\- Service providers of cancer care at hospital research site",{"count":216,"type":19},50,"Introduction:\n\nResearch has routinely been conducted into the experiences and well-being of children and young people with cancer and their families. However, there is little research that directly involves those with learning disabilities (LD) and or who are autistic. This is despite some cancers being more far more prevalent in some syndromes associated with learning disabilities, for example Downs Syndrome. More generally paediatric hospital care, recent research has highlighted inequity for children with LD, compared with children and young people without LD. Staff often feel less capable and confident to deliver care to those with learning disabilities, as well as having less capacity. Still less is known about cancer care for autistic children and young people, for example relating to symptom management. We aim to explore how inequity might present in cancer care for children and young people aged 5-15 years with and without LD and or who are autistic to see what inequities exist, for whom, why and in what circumstances.\n\nMethods and analysis:\n\nA transformative mixed methods design will be used, comprising an individual staff and organisational level survey, retrospective case note review, ethnographic observations of clinical care, family and staff interviews, and participatory workshops. The ethnographer will follow and observe individual children and their families. A 'toolbox' of creative participatory methods will be employed, including providing a co-designed research data collection journal to support elicitation of the child's perspective.\n\nEthics and Dissemination: The study will run from September 2024 to January 2026. Health Research Authority approval is granted (REC Reference no. 24\u002FLO\u002F0410 \\| IRAS Project ID: 335623) for work package 2 and 3 involving the ethnography, with interviews and workshops.",[219,220,221],"Cancer","Oncology","Haematological Malignancy",{"date":223,"type":36},"2024-07-01",{"date":225,"type":19},"2024-09-02",{"date":227,"type":19},"2026-03-01",{"name":42,"class":43},{"id":230,"slug":231,"hasResults":11,"nctId":232,"briefTitle":233,"officialTitle":234,"acronym":4,"eligibilityCriteria":235,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":129,"enrollmentInfo":236,"targetDuration":4,"studyType":20,"phases":237,"briefSummary":238,"conditions":239,"keywords":244,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":252,"lastUpdatePostDateStruct":253,"startDateStruct":255,"completionDateStruct":257,"leadSponsor":259,"locationsCount":68},"100548446","micro-dosing-indocyanine-green-icg-in-children-100548446","NCT06421103","Micro-dosing Indocyanine Green (ICG) in Children","Investigating Micro-dosing of Indocyanine Green (ICG) for Intraoperative Perfusion Assessment in Children.","Inclusion Criteria:\n\n* Children aged ≤18 years of age\n* Elective surgery including gastrointestinal or genitourinary tract perfusion assessment\n\nExclusion Criteria:\n\n* Renal and liver dysfunction\n* Active infection\n* Coagulopathy\n* Complex congenital heart defect\n* Previous documented allergy to ICG injection or other iodinated contrast agents\n* Patients suffering from hyperthyroidism or autonomic thyroid adenomas\n* Premature infants or neonates in whom exchange transfusion is indicated, due to the hyperbilirubinaemia risk\n* Concurrent use of sodium bisulphite-containing preparations, such as certain heparin preparations",{"count":192,"type":19},[22],"The goal of this cohort study is to identify the lowest dose of Indocyanine Green (ICG) that achieves satisfactory intra-operative fluorescence for the assessment of gastrointestinal or genitourinary tract perfusion in children.",[240,241,242,243],"Perfusion","Fluorescence Imaging","Gastrointestinal Tract Disorders","Genitourinary Disease",[245,246,247,248,249,250,251],"Paediatric Surgery","Pediatric Surgery","Fluorescence","Imaging","Fluorescence Guided Surgery (FGS)","Intra-operative perfusion assessment","Indocyanine Green","2024-05-15",{"date":254,"type":36},"2024-05-20",{"date":256,"type":19},"2024-06",{"date":258,"type":19},"2028-08",{"name":42,"class":43},{"id":261,"slug":262,"hasResults":11,"nctId":263,"briefTitle":264,"officialTitle":265,"acronym":4,"eligibilityCriteria":266,"healthyVolunteers":11,"sex":267,"minAge":268,"maxAge":269,"enrollmentInfo":270,"targetDuration":4,"studyType":20,"phases":272,"briefSummary":274,"conditions":275,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":277,"lastUpdatePostDateStruct":278,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":68},"100331901","phase-1-lentiviral-gene-therapy-for-x-linked-severe-combined-immunodeficiency-100331901","NCT03601286","Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency","Phase I\u002FII Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan","Inclusion Criteria:\n\n1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \\\u003C10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG\n2. Lack of an HLA identical (A, B, C, DR, DQ) related donor\n3. Age \\\u003C5 years\n4. Signed informed consent\n5. Documentation of willingness to follow up for 15 years post-infusion\n6. If the patient has previously undergone allogeneic transplant or gene therapy, insufficiency of graft-derived T cell engraftment must be documented.\n7. Age at least 8 weeks of age by the time of busulfan administration\n\nExclusion Criteria:\n\n1. Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below).\n\n   1. Mechanical ventilation including continuous positive airway pressure\n   2. Abnormal liver function defined by AST and ALT \\>10 times the upper range of normal OR Bilirubin \\>2 mg\u002FdL\n   3. Shortening fraction on echocardiogram \\\u003C25% or ejection fraction \\\u003C50%\n   4. Renal failure defined as glomerular filtration rate \\\u003C30 ml\u002Fmin\u002F1.73 m2 or dialysis dependence\n2. Uncontrolled seizure disorder\n3. Encephalopathy\n4. Documented coexistence of any disorder known to affect DNA repair\n5. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease\n6. Patients with evidence of infection with HIV-1\n7. Previous allogeneic transplant with cytoreductive chemotherapy\n8. Major (life-threatening) congenital anomalies. Examples of \"major (life-threatening) congenital anomalies\" include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other major central nervous system malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function.\n9. Other conditions which in the opinion of the P.I. or Co-investigators, contra-indicate collection and\u002For infusion of transduced cells or indicate patient's inability to follow the protocol. These may include for example clinical ineligibility to receive anaesthesia, severe deterioration of clinical condition of the patient after collection of bone marrow but before infusion of transduced cells, or documented refusal or inability of the family to return for scheduled visits. There may be other unforeseen rare circumstances that would result in exclusion of the patient, such as sudden loss of legal guardianship.","MALE","8 Weeks","5 Years",{"count":271,"type":19},5,[273],"PHASE1","Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. SCID arises from a variety of molecular defects which affect lymphocyte development and function. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. SCID-X1 is caused by defects in the common cytokine receptor gamma chain, which was originally identified as a component of the high affinity interleukin-2 receptor (IL2RG).\n\nAllogeneic haematopoietic stem cell transplantation (HSCT), which replaces the patient's bone marrow with that of a healthy donor, is the only treatment that definitively restores the normal function of the bone marrow. HSCT is the first choice of treatment for patients with signs of bone marrow failure and a fully-matched related donor. However, patients without a fully-matched related donor have much worse overall outcomes from HSCT.\n\nThis study will investigate whether patients with SCID-X1 without a fully matched related donor may benefit from gene therapy. To do this the investigators propose to perform a phase I\u002FII clinical trial to evaluate the safety and efficacy (effect) of gene therapy for SCID-X1 patients using a lentivirus delivery system containing the IL2RG gene. Up to 5 eligible SCID-X1 patients will undergo mobilisation and harvest of their haematopoietic stem precursor cells (HPSCs). In the laboratory the disabled lentivirus will be used to insert a normal human IL2RG gene into the patient's harvested HPSCs. Patients will receive chemotherapy conditioning prior to cell infusion, in order to enhance grafting. The genetically corrected stem cells will then be re-infused into the patient. Patients will be followed up for 2 years. This trial will determine whether gene therapy for SCID-X1 using a lentiviral vector is safe, feasible and effective",[276],"Severe Combined Immunodeficiency, X-Linked","2023-10-11",{"date":279,"type":36},"2023-10-12",{"date":281,"type":36},"2018-12-21",{"date":283,"type":19},"2026-08",{"name":42,"class":43},{"id":286,"slug":287,"hasResults":11,"nctId":288,"briefTitle":289,"officialTitle":290,"acronym":4,"eligibilityCriteria":291,"healthyVolunteers":11,"sex":16,"minAge":292,"maxAge":4,"enrollmentInfo":293,"targetDuration":4,"studyType":20,"phases":294,"briefSummary":296,"conditions":297,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":299,"lastUpdatePostDateStruct":300,"startDateStruct":302,"completionDateStruct":304,"leadSponsor":306,"locationsCount":68},"100455216","phase-1-lentiviral-gene-therapy-for-p47-ar-cgd-100455216","NCT05207657","Lentiviral Gene Therapy for p47 AR-CGD","Phase I\u002FII, Non-randomised, Single-centre, Open-label Study of pCHIM-p47 (Lentiviral Vector Transduced CD34+ Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease","Inclusion Criteria:\n\n1. p47 AR-CGD patients \\> 23 months of age\n2. Molecular diagnosis confirmed by Deoxyribonucleic acid (DNA) sequencing and supported by laboratory evidence for absent or reduction \\> 95% of the biochemical activity of the NAHPD-oxidase\n3. At least one prior, ongoing or refractory severe infection and\u002For inflammatory complications requiring hospitalisation despite conventional therapy\n4. No 10\u002F10 human leukocyte antigen (HLA)-matched donor available after initial search of National Marrow Donor Program (NMDP) registries performed within the last year\n5. No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV ribonucleic acid (RNA) positive), Cytomegalovirus (CMV), adenovirus, parvovirus B 19 or toxoplasmosis\n6. Written informed consent for adult patient\n7. Parental\u002Fguardian and, where appropriate, child's signed consent\u002Fassent\n\nExclusion Criteria:\n\n1. Age ≤ 23 months or \\> 35 kg body weight\n2. 10\u002F10 HLA identical (A,B,C,DR,DQ) family or unrelated adult donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure\n3. Contraindication for leukapheresis (Haemoglobin \\\u003C8g\u002Fdl, cardiovascular instability, severe coagulopathy)\n4. Appropriate organ function as outlined below must be observed within 8 weeks of entering this trial.\n\n   a) Haematologic i) Anaemia (hemoglobin \\\u003C 8 g\u002Fdl). ii) Neutropenia (absolute granulocyte count \\\u003C1,000\u002Fmm3 iii) Thrombocytopenia (platelet count \\\u003C 150,000\u002Fmm3). iv) Prothrombin Time (PT) or Partial thromboplastin time (PTT) \\> 2 X the upper limits of normal (ULN) (patients with a correctable deficiency controlled on medication will not be excluded).\n\n   v) Cytogenetic abnormalities known to be associated with haematopoietic defect on peripheral blood or bone marrow.\n\n   b) Infectious i) Evidence of infection with HIV-1 and -2, hepatitis B, Hepatitis C, adenovirus, parvovirus B 19 or toxoplasmosis within 8 weeks prior to mobilisation\u002Fpheresis or bone marrow harvest. CMV infection is allowable as long as the infection is under control.\n\n   c) Pulmonary i) Resting O2 saturation by pulse oximetry \\\u003C 90% on room air. d) Cardiac i) Abnormal electrocardiogram (ECG) indicating cardiac pathology. ii) Uncorrected congenital cardiac malformation with clinical symptomatology. iii) Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, iv) Hypotension. v) Poor cardiac function as evidenced by Left Ventricular Ejection Fraction (LVEF) \\\u003C 40% on echocardiogram.\n\n   e) Neurologic i) Significant neurologic abnormality by examination. ii) Uncontrolled seizure disorder. f) Renal i) Renal insufficiency: serum creatinine greater than or equal to 1.5 mg\u002Fdl, or greater than or equal to 3+ proteinuria ii) Abnormal serum sodium, potassium, calcium, magnesium at grade III or IV according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 g) Hepatic\u002FGI: i) Serum transaminases \\> 5X the upper limit of normal (ULN). ii) Serum Bilirubin \\> 2X ULN. iii) Serum Glucose \\> 1.5x ULN. h) Oncologic i) Evidence of active malignant disease\n5. General\n\n   1. Expected survival \\\u003C 6 months.\n   2. Major congenital anomaly.\n   3. Ineligible for autologous Haematopoietic Stem Cell Transplant (HSCT) by the criteria at the clinical site.\n   4. Contraindication for administration of conditioning medication\n   5. Known sensitivity to Busulfan\n6. Administration of gamma-interferon within 30 days before the infusion of transduced, autologous CD34+ cells\n7. Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period\n8. Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful study completion\n9. Patient\u002FParent\u002FGuardian unable or unwilling to comply with the protocol requirements.","23 Months",{"count":271,"type":19},[273,295],"PHASE2","Chronic Granulomatous Disease (CGD) is a rare inherited disorder in which patients suffer from severe infection and inflammation. The first indications of disease usually appear in early childhood. The basic defect has been found to be lie in specialised white blood cells called phagocytic cells, which are responsible for engulfing and destroying germs. In CGD, there is a defect in an enzyme (known as the NADPH-oxidase) that is responsible for generating bleach like substances that are important for killing some important germs. In one form of the disease known as p47 AR-CGD (which accounts for 30% of patients), there are defined mistakes in a gene called NCF1. This gene is needed to form a key component of NADPH-oxidase.\n\nIn many cases, patients can be protected from infection by constant intake of antibiotics. However, in others potentially life-threatening infections break through. In some cases patients also develop serious inflammation requiring high doses of drugs such as steroids. CGD can be cured by bone marrow transplant and the best results are available when a matched sibling donor is available. Transplant from unmatched donors have a much worse outcome and as a result alternative treatments for patients without a matched donor are highly desirable.\n\nGene therapy of p47 AR-CGD is performed by introducing a normal copy of the human NCF-1 gene into the blood forming stem cells in the patients' bone marrow by using a gene carrier (in this study called a lentiviral vector). After treatment of the bone marrow cells in a specialised laboratory they are given back to the patient and will grow into functional phagocytic cells. There have been no previous clinical trials for patients with p47 AR-CGD however there have been previous gene therapy clinical trials conducted in the UK for patients with the most common form of CGD, known as X-CGD.",[298],"P47-Phox, Deficiency of","2023-05-14",{"date":301,"type":36},"2023-05-16",{"date":303,"type":36},"2023-03-20",{"date":305,"type":19},"2029-04-01",{"name":42,"class":43},""]