[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"immune-thrombocytopenic-purpura--itp-\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:immune-thrombocytopenic-purpura--itp-":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,50,84],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":4,"studyType":24,"phases":25,"briefSummary":27,"conditions":28,"keywords":31,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100645085","model-early-immunologic-stages-of-pediatric-hematological-pre-lupus-100645085",false,"NCT07680010","Model Early Immunologic Stages of Pediatric Hematological Pre-lupus","Model Early Immunologic Stages of Pediatric Hematological Pre-lupus in Order to Prevent SLE in Children.","PRELUDE","* Inclusion criteria:\n\n  * For patients :\n\n    * Child or adolescent with newly diagnosed ITP or SLE according to the specific definitions of ITP or SLE, prior to any treatment,\n    * Over 1 and under 18 years of age at diagnosis, weighing more than 7 kg.\n    * Written consent from parents or guardians,\n    * Patient affiliated to a social security scheme.\n  * For controls :\n\n    * Over 1 and under 18 years of age at diagnosis, weighing more than 7 kg.\n    * Follow-up in the day hospital at the Bordeaux University Hospital, for a condition that does not affect the immune system\n    * Matched on age,\n    * Written consent from parents or guardians,\n    * Patient affiliated to a social security scheme\n* Exclusion criteria:\n\n  * For patients :\n\n    * ITP secondary to a known cause: previous or concomitant immune deficiency, bone marrow or organ transplantation, other autoimmune disease, Evans syndrome (autoimmune hemolytic anemia or autoimmune neutropenia present at ITP diagnosis) or cancer with immunosuppressive therapy.\n    * Treatment with immunomodulation or immunosuppressants (including immunoglobulins, corticoids, hydroxychloroquine), started prior to inclusion (day of sampling).\n    * Pregnant women, women in labour and breastfeeding women\n  * For controls :\n\n    * Suffering from an immunological disease,\n    * Infection within fifteen days prior to inclusion,\n    * Immunomodulatory therapy.\n    * Pregnant women, women in labour and breastfeeding women",true,"ALL","1 Year","18 Years",{"count":22,"type":23},105,"ESTIMATED","INTERVENTIONAL",[26],"NA","Immunologic thrombocytopenic purpura (ITP) in children is a pre-lupus condition if associated with the presence of anti-nuclear antibodies (ANA), providing a unique model for understanding the natural history of autoimmunity, particularly that of systemic lupus erythematosus (SLE). We will describe the shared and\u002For unique immunological pathways involved at diagnosis in 70 children with ITP and in 20 children with SLE, and compare them between ITP-ANA- (more often transient), ITP-ANA+ (pre-lupus condition, more often persistent) and SLE",[29,30],"Immune Thrombocytopenic Purpura ( ITP )","Systemic Lupus Erythematosus (SLE)",[32,33,34,35,36],"Children","Immunologic Thrombocytopenic Purpura","Anti-Nuclear Antibody","Systemic Lupus Erythematosus","Biomarkers","NOT_YET_RECRUITING","2026-06-25",{"date":40,"type":41},"2026-07-01","ACTUAL",{"date":43,"type":23},"2026-07",{"date":45,"type":23},"2033-10",{"name":47,"class":48},"University Hospital, Bordeaux","OTHER",2,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":4,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":18,"minAge":57,"maxAge":58,"enrollmentInfo":59,"targetDuration":4,"studyType":24,"phases":61,"briefSummary":64,"conditions":65,"keywords":4,"overallStatus":72,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":83},"100607922","phase-2-a-study-of-efgartigimod-iv-in-participants-from-12-years-to-less-than-18-years-of-age-with-chronic-immune-thrombocytopenia-itp-100607922","NCT07194850","A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)","A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP","Inclusion Criteria:\n\n* Is aged 12 to less than 18 years when completing the informed consent process\n* Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete\n* Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.\n* Has documented prior response, defined as 1 platelet count of ≥50 × 10\\^9\u002FL to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin\n* Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy\n* Has documented mean platelet count of less than 30 x10\\^9\u002FL\n\nExclusion Criteria:\n\n* Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP\n* Nonimmune thrombocytopenia\n* ITP-associated critical or severe bleeding\n* History of hereditary thrombocytopenia","12 Years","17 Years",{"count":60,"type":23},24,[62,63],"PHASE2","PHASE3","The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP).\n\nThe study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.\n\nMore information can be found here: https:\u002F\u002Fclinicaltrials.argenx.com\u002Fadvancejunior",[66,67,68,69,29,70,71],"Immune Thrombocytopenia (ITP)","ITP - Immune Thrombocytopenia","ITP","Immune Thrombocytopenic Purpura","Idiopathic Thrombocytopenic Purpura","Idiopathic Thrombocytopenic Purpura (ITP)","RECRUITING","2026-06-11",{"date":75,"type":41},"2026-06-12",{"date":77,"type":41},"2025-10-20",{"date":79,"type":23},"2030-10",{"name":81,"class":82},"argenx","INDUSTRY",10,{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":89,"acronym":4,"eligibilityCriteria":90,"healthyVolunteers":11,"sex":18,"minAge":91,"maxAge":92,"enrollmentInfo":93,"targetDuration":95,"studyType":96,"phases":4,"briefSummary":97,"conditions":98,"keywords":104,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":112,"lastUpdatePostDateStruct":113,"startDateStruct":115,"completionDateStruct":117,"leadSponsor":119,"locationsCount":121},"100583234","validation-of-the-eq-5d-y-3l-and-eq-5d-y-5l-for-paediatric-patients-in-china-100583234","NCT06873672","Validation of the EQ-5D-Y-3L and EQ-5D-Y-5L for Paediatric Patients in China","Using EQ-5D-Y to Measure the Health of Patients for Multiple Paediatric Health Conditions: a Project to Validate the Self-complete, Interviewer-administered and Proxy Administered Versions of EQ-5D-Y-3L and EQ-5D-Y-5L in China","Inclusion Criteria:\n\n* Children (Patients)\n\n  1. Age: 5-16 years old.\n  2. Diagnosis: Clinically confirmed diagnosis of one of the following conditions:\n\n     Pneumonia (hospitalized due to community-acquired pneumonia); Central Nervous System (CNS) Solid Tumours (receiving active treatment or in post-treatment follow-up); Immune Thrombocytopenic Purpura (ITP) (diagnosed with acute or chronic ITP).\n  3. Health Status: Cognitively and physically capable of completing HRQoL assessments (for self-complete versions).\n  4. Language Proficiency: Able to understand and communicate in Mandarin Chinese.\n  5. Consent: Written informed consent obtained from the primary caregiver and verbal or written assent from the child (if age-appropriate).\n* Caregivers (Parents\u002FFamily Caregivers)\n\n  1. Primary Caregiver: Must be a parent or a long-term family caregiver (e.g., grandparent, older sibling, aunt, or uncle).\n  2. Involvement: Directly responsible for the daily care and medical decision-making of the child.\n  3. Language Proficiency: Able to understand and complete both the self-complete and proxy-reported HRQoL assessments in Mandarin Chinese.\n  4. Consent: Willing to provide informed consent for participation.\n\nExclusion Criteria:\n\n* Children (Patients)\n\n  1. Severe Cognitive or Developmental Impairment: Diagnosed with intellectual disability, autism spectrum disorder, or any neurological condition that prevents understanding and completing HRQoL assessments.\n  2. Severe Physical Disability:\n\n     Conditions that make it impossible to respond to surveys (e.g., advanced neuromuscular disease, severe paralysis).\n  3. Comorbidities: Presence of another major chronic illness that could significantly affect HRQoL measurement (e.g., severe congenital heart disease, uncontrolled epilepsy).\n  4. Recent Participation in Similar Studies:\n\n     If the child has participated in another HRQoL validation study within the past 6 months.\n  5. Inability to Complete Study Follow-Up: Expected to be transferred to another hospital or relocated during follow-up.\n* Caregivers (Parents\u002FFamily Caregivers)\n\n  1. Not the Primary Caregiver:\n  2. Individuals who do not have daily caregiving responsibilities for the child.\n\n     Cognitive Impairment or Severe Health Conditions:\n  3. Caregivers with severe mental illness, dementia, or a medical condition that affects their ability to complete surveys.\n  4. Language Barrier: Unable to understand and complete HRQoL assessments in Mandarin Chinese.\n  5. Refusal to Participate: Caregivers who decline to provide informed consent.","5 Years","16 Years",{"count":94,"type":23},360,"2 Weeks","OBSERVATIONAL","The goal of this observational study is to validate the EQ-5D-Y-3L and EQ-5D-Y-5L in measuring health-related quality of life (HRQoL) for paediatric patients with different health conditions in China. The main research questions focus on comparing the psychometric performance of EQ-5D-Y-3L and EQ-5D-Y-5L, evaluating the differences between self-complete (SC), interviewer-administered (IA), and proxy-administered (Proxy) versions, and assessing their validity against other HRQoL measures like PedsQL. The study will recruit 360 inpatient-caregiver dyads from three disease groups, i.e., pneumonia, paediatric central nervous system (CNS) solid tumours, and immune thrombocytopenic purpura (ITP), using cross-sectional and longitudinal surveys. Baseline assessments will be conducted at hospital admission, with follow-up at hospital discharge (within 14 days). A qualitative component will explore acceptability and interpretation of EQ-5D-Y. The findings will contribute to the methodological development of EQ-5D-Y, with potential implications for HRQoL measurement and economic evaluations in paediatric healthcare.",[99,100,101,102,103,29],"Quality of Life (QOL)","Patient-Reported Outcome Measures (PROMs)","Pneumonia Childhood","Neuroblastoma (NB)","Wilms Tumour",[105,106,107,108,109,110,111],"Quality of life","children","psychometric measurements","Paediatric pneumonia","Paediatric central nervous system solid tumour","paediatric immune thrombocytopenic purpura","spillover effect","2025-03-07",{"date":114,"type":41},"2025-03-13",{"date":116,"type":23},"2025-04-01",{"date":118,"type":23},"2027-12-31",{"name":120,"class":48},"RenJi Hospital",1]