[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neuroblastoma-nb\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neuroblastoma-nb":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,40,73,100,132,167],{"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":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100617454","phase-2-phase-ii-study-of-chidamide-dinutuximab-beta-irinotecan-temozolomide-for-refractoryrelapsed-neuroblastoma-in-children-100617454",false,"NCT07318831","Phase II Study of Chidamide-Dinutuximab Beta-Irinotecan-Temozolomide for Refractory\u002FRelapsed Neuroblastoma in Children","A Phase II Trial of Chidamide Combined With Dinutuximab Beta, Irinotecan, and Temozolomide for Refractory or Relapsed Neuroblastoma in Children","Inclusion Criteria:\n\n1. Patients with histologically diagnosed neuroblastoma, defined according to the International Neuroblastoma Risk Group (INRG) classification system or the Chinese expert consensus\u002Fguideline for pediatric neuroblastoma.\n2. Patients with relapsed or refractory neuroblastoma. Relapsed: any patient with recurrent neuroblastoma. Refractory: patients showing an inadequate response (partial response, minor response, or stable disease) to prior therapy, leading to progression.\n3. Prior treatment with epigenetic drugs (e.g., HDAC inhibitors, DNA methylation inhibitors) or GD2 monoclonal antibodies does not affect eligibility for this study.\n4. Presence of evaluable disease.\n5. Performance Status: Lansky score ≥50%, Karnofsky score ≥50%, or ECOG score ≤3.\n6. Life expectancy ≥12 weeks.\n7. Bone marrow function: Without bone marrow disease: Platelets ≥75×10⁹\u002FL, Absolute Neutrophil Count (ANC) ≥0.75×10⁹\u002FL, Hemoglobin ≥8 g\u002FdL (transfusion allowed). With bone marrow disease: Platelets ≥50×10⁹\u002FL, ANC ≥0.5×10⁹\u002FL, Hemoglobin ≥8 g\u002FdL (transfusion allowed).\n8. Renal function: No clinically significant proteinuria (morning urine dipstick \\\u003C2+). If proteinuria ≥2+ is detected, the protein-to-creatinine (Pr\u002FCr) ratio must be \\\u003C0.5 or 24-hour protein excretion must be \\\u003C0.5 g.\n9. Serum creatinine ≤1.5 × ULN; if higher, the calculated glomerular filtration rate (by radioisotope method) must be ≥60 mL\u002Fmin\u002F1.73 m².\n10. Hepatic function: AST or ALT ≤2.5 × ULN and total bilirubin ≤1.5 × ULN. In the presence of liver metastases: AST or ALT ≤5 × ULN and total bilirubin ≤2.5 × ULN.\n11. Cardiac function: Left ventricular shortening fraction ≥29% on echocardiogram.\n12. Coagulation: For patients not on anticoagulation therapy: INR ≤1.5 and APTT ≤1.5 × ULN. Anticoagulation is allowed if INR or APTT is within the therapeutic range (per institutional standards) and the patient has been on a stable dose for at least two weeks prior to study enrollment.\n13. Oxygen saturation \\>94% on room air.\n14. Ability to comply with the study visit schedule and other protocol requirements.\n\nExclusion Criteria:\n\n1. Patients with CTCAE v5.0 Grade 3 or higher toxicities involving hearing impairment, hematologic disorders, hepatic, or renal diseases.\n2. Patients with CTCAE v5.0 Grade 2 or higher neurotoxicity.\n3. Major surgical procedure within 14 days prior to the first dose of the study drug.\n4. Severe infection (requiring IV antibiotics, antifungals, or antivirals) within one week prior to treatment, or unexplained fever \\>38.5°C during screening or before the first dose.\n5. Congenital or acquired immunodeficiency, or active infectious diseases such as HIV or active hepatitis (with transaminase levels not meeting inclusion criteria; HBV DNA ≥1000 IU\u002FmL; HCV RNA ≥1000 IU\u002FmL). Chronic HBV carriers with HBV DNA \\\u003C2000 IU\u002FmL may be enrolled if they receive concurrent antiviral therapy during the trial.\n6. Any concomitant condition that, in the investigator's judgment, seriously jeopardizes patient safety, may confound the study results, or could impede the patient's completion of the study.","ALL",{"count":18,"type":19},27,"ESTIMATED","INTERVENTIONAL",[22],"PHASE2","This is a Phase II clinical trial investigating the effectiveness and safety of a four-drug combination-Chidamide, Dinutuximab Beta, Irinotecan, and Temozolomide-for children with relapsed or refractory neuroblastoma. The primary goal is to evaluate how well this regimen works to control the cancer, while the secondary goal is to closely monitor its safety and side effects in these young patients.",[25,26],"Neuroblastoma (NB)","Neuroblastoma in Children","RECRUITING","2026-06-02",{"date":30,"type":31},"2026-06-04","ACTUAL",{"date":33,"type":31},"2026-01-06",{"date":35,"type":19},"2028-12-31",{"name":37,"class":38},"Tianjin Medical University Cancer Institute and Hospital","OTHER",1,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":46,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":16,"minAge":48,"maxAge":49,"enrollmentInfo":50,"targetDuration":4,"studyType":20,"phases":52,"briefSummary":54,"conditions":55,"keywords":57,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":39},"100621818","phase-3-chemoimmunotherapy-combined-with-autologous-nk-cell-therapy-for-pediatric-patients-with-refractory-and-relapsed-high-risk-neuroblastoma-and-ganglioneuroblastoma-100621818","NCT07375563","Chemoimmunotherapy Combined With Autologous NK Cell Therapy for Pediatric Patients With Refractory and Relapsed High-Risk Neuroblastoma and Ganglioneuroblastoma","Chemoimmunotherapy Combined With Autologous NK Cell Therapy for the Treatment of Pediatric Patients With Refractory and Relapsed High-Risk Neuroblastoma and Ganglioneuroblastoma","NB-NK-2026","Inclusion Criteria:\n\n* Signed voluntary informed consent to participate in the clinical trial\n* Histologically verified diagnosis of neuroblastoma or ganglioneuroblastoma\n* Patients stratified to the high-risk group according to the criteria of the German Society of Pediatric Oncology and Hematology (GPOH) - NB 2004, aged from 18 months to 18 years, and meeting the following conditions:\n\n  1. Arm A: Refractory disease - patients who have completed the induction phase of therapy (6 cycles of N5\u002FN6) with a poor response to therapy (MR, SD), with the exception of PD\n  2. Arm В: Relapsed\u002Fprogressive disease - patients who develop any new tumor lesions (after having previously achieved СR), or any new tumor lesion; an increase of \\>25% in any previously existing measurable lesion; or newly detected bone marrow involvement by NB cells in cases where the bone marrow had previously been free of involvement\n* Performance status ≥ 70% (Lansky or Karnofsky scale) at the time of determining the indication for chemoimmunotherapy combined with NK cell therapy.\n* Expected life expectancy ≥ 12 weeks.\n* No signs of drug-induced neuropathy or neuropathic pain.\n* Adequate liver function: alanine aminotransferase (ALT) \u002F aspartate aminotransferase (AST) activity \\\u003C 5 × upper limit of normal (ULN).\n* Adequate renal function: creatinine clearance or glomerular filtration rate (GFR) \\> 60 mL\u002Fmin\u002F1.73 m².\n* Coagulation parameters: prothrombin index (PTI) 70-120%; activated partial thromboplastin time (APTT) \\\u003C 36 seconds.\n* No clinical signs of heart failure; left ventricular ejection fraction (LVEF) ≥ 55%.\n* Adequate respiratory function (oxygen saturation by pulse oximetry \\> 94% on room air, no dyspnea at rest), and no pathological findings on chest X-ray.\n* Completion of comprehensive assessment to evaluate the extent of the tumor process.\n\nExclusion Criteria:\n\n* Lack of a signed voluntary informed consent form for participation in the clinical study.\n* Absence of comprehensive pre-treatment assessment results at the time of initiation of specific therapy.\n* Patients with NBL or ganglioneuroblastoma stratified to low or intermediate-risk group\n* Good response (PR, VGPR, CR) or PD at the end of the induction phase of therapy (applicable only to patients receiving therapy within the framework of the intensified induction phase).\n* Progressive or relapsed disease with central nervous system involvement and\u002For leptomeningeal involvement.\n* History of acute intolerance reactions to the main chemotherapeutic and immunobiological agents and supportive care drugs used in this clinical trial protocol.\n* Presence of complications of the underlying disease and comorbidities that preclude treatment within this protocol, including severe type I hypersensitivity reactions in the medical history.\n* Requirement for concomitant medications with known cross pharmacodynamic interactions with the drugs used in this clinical trial protocol.\n* Presence of ultrasonographic signs of heart failure (LVEF ≤ 55%), clinical and laboratory signs of chronic kidney disease of stage ≥ III, or kidney injury of grade I, F or L according to the standardized RIFLE criteria for acute kidney injury (an acronym for \"risk, injury, failure, loss, end-stage\").\n* Pregnancy, due to the high teratogenicity and toxicity of the drugs used in this clinical trial protocol. Female patients of childbearing potential are required to undergo pregnancy testing.\n* Mental illness of the patient or legal guardians that makes it impossible to understand the nature of the study and compromises adherence to medical prescriptions and sanitary-hygienic requirements.","18 Months","18 Years",{"count":51,"type":19},5,[53],"PHASE3","Neuroblastoma (NB) is a malignant neoplasm of the sympathetic nervous system, occurring in 1 in 8,000 live births, accounting for 6-10% of all childhood malignant neoplasms and responsible for 12-15% of mortality -, making it the most common and life-threatening extracranial tumor in childhood.\n\nPatients with stage 4 high-risk NB is the subgroup with the poorest prognosis. Within this group, two subgroups with an extremely unfavorable disease course are distinguished: patients with a poor response to the induction phase of therapy (refractory disease) and patients with relapsed or progressive disease.\n\nNowadays, 10-15% of patients show a poor end-induction response, whereas achieving a good end-induction response associated with better long-term survival. Improvement of the response to induction therapy may contribute to better treatment outcomes in newly diagnosed high-risk NB patients and can be achieved by intensification of the induction phase to decrease the number of patients with refractory disease. Also intensification of the second-line therapy may contribute to better responses in patients with relapsed and progressive disease.\n\nProtocol aimed to overcome heterogeneous tumor drug resistance through the synergistic interaction of cytostatic and immunobiological agents in combination with NK cell therapy.\n\nThis approach combines cytotoxic agents with anti-GD2 monoclonal antibodies (mAb) to enhance antitumor activity. Cultured, ex vivo-activated autologous NK cells are infused to compensate for effector cell depletion during therapy and to augment antibody-dependent cellular cytotoxicity (ADCC), potentially improving clinical outcomes.\n\nThis comprehensive approach opens novel prospects for enhancing treatment efficacy in patients with refractory and relapsed high-risk NB.\n\nThe expected outcomes of this protocol include a significant increase in therapeutic efficacy indicators - objective response rate (ORR), overall survival (OS), progression-free survival (PFS) and relapse-free survival (RFS), as well as in patient quality of life.",[25,56],"Ganglioneuroblastoma",[58,59,60,61,62,63],"Refractory\u002Frelapsed neuroblastoma","ganglioneuroblastoma","children","autologous NK cells","chemoimmunotherapy","dinutuximab beta","2026-01-21",{"date":66,"type":31},"2026-01-29",{"date":68,"type":31},"2025-11-19",{"date":70,"type":19},"2028-11-19",{"name":72,"class":38},"Federal Research Institute of Pediatric Hematology, Oncology and Immunology",{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":77,"acronym":78,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":49,"enrollmentInfo":80,"targetDuration":82,"studyType":83,"phases":4,"briefSummary":84,"conditions":85,"keywords":86,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":39},"100597919","clinical-application-study-of-123imetaiodobenzylguanidine-and-somatostatin-receptor-targeted-imaging-in-the-diagnosis-and-staging-of-neuroblastoma-100597919","NCT07064746","Clinical Application Study of [123I]Metaiodobenzylguanidine and Somatostatin Receptor-Targeted Imaging in the Diagnosis and Staging of Neuroblastoma","MIBG & SSTR NB","Inclusion Criteria:\n\n1. Ages 0-18 years, any sex\n2. Histologically confirmed or clinically highly suspected neuroblastoma with planned imaging assessment\n3. At least one measurable lesion on conventional imaging such as CT or MRI\n4. ECOG performance status 0-2 and an expected survival of ≥3 months\n5. Hematologic, hepatic, and renal function meeting the following criteria: Hb ≥ 90 g\u002FL, ANC ≥ 1.5 × 10⁹\u002FL, platelets ≥ 100 × 10⁹\u002FL, ALT\u002FAST ≤ 2.5 × ULN, Cr ≤ 1.5 × ULN\n6. Informed consent signed by the subject or legal guardian\n\nExclusion Criteria:\n\n1. Known history of severe allergy to the radioactive isotope or molecular probe components to be used;\n2. Pregnancy or lactation;\n3. Receipt of high-dose chemotherapy\u002Fradiotherapy, radionuclide therapy, or participation in other interventional clinical trials within the past 4 weeks;\n4. Severe or uncontrolled cardiac, pulmonary, hepatic, or renal insufficiency, or active severe infection;\n5. Bone marrow suppression not yet recovered (Hb \\\u003C 90 g\u002FL, ANC \\\u003C 1.5 × 10⁹\u002FL, platelets \\\u003C 100 × 10⁹\u002FL);\n6. Inability to complete imaging examinations such as PET\u002FCT (e.g., severe claustrophobia, inability to lie supine for ≥ 30 min);\n7. Any other situation deemed inappropriate for enrollment by the investigator.",{"count":81,"type":19},150,"6 Months","OBSERVATIONAL","The goal of this prospective observational study is to evaluate the diagnostic performance of ¹²³I-MIBG SPECT\u002FCT and SSTR PET in Chinese children with suspected or confirmed neuroblastoma (NB). The main questions it aims to answer are:\n\n* What are the sensitivity and specificity of each modality-alone and combined-for initial staging and for detecting relapse or metastasis?\n* Can SSTR-targeted PET reliably identify MIBG-negative NB lesions and help select candidates for peptide-receptor radionuclide therapy (PRRT)?\n\nPediatric patients (≤18 years) undergoing routine evaluation for NB will receive both imaging studies; results will be correlated with histopathology, clinical course, and 24-month follow-up.",[25],[87,88,89,90],"neuroblastoma","SSTR","MIBG","PET","2025-07-17",{"date":93,"type":31},"2025-07-21",{"date":95,"type":31},"2022-07-10",{"date":97,"type":19},"2027-07-10",{"name":99,"class":38},"Nanjing First Hospital, Nanjing Medical University",{"id":101,"slug":102,"hasResults":11,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":4,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":16,"minAge":107,"maxAge":108,"enrollmentInfo":109,"targetDuration":4,"studyType":20,"phases":111,"briefSummary":113,"conditions":114,"keywords":116,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":39},"100567905","phase-1-safety-and-efficacy-of-systemic-allogenic-nk-cells-in-rr-neuroblastoma-100567905","NCT06674265","Safety and Efficacy of Systemic Allogenic NK Cells in R\u002FR Neuroblastoma","Evaluation of Safety and Efficacy Evaluation Post Intravenous Infusion of Activated NK Cells in Recurrent and Refractory High-risk Neuroblastoma Patients","Inclusion Criteria:\n\n1. High-risk neuroblastoma that is resistant to standard induction therapy based on COG (Children's Oncology Group) criteria (according to INRG criteria and having received at least 4 cycles of multi-drug induction chemotherapy, and not responding to conventional treatments).\n2. Evidence of relapse or progression of neuroblastoma after autologous peripheral blood stem cell transplantation or aggressive therapy.\n3. A minimum life expectancy of 6 months.\n4. Patients must have a pathological diagnosis of neuroblastoma and\u002For confirmation of tumor cells in the bone marrow with increased urinary catecholamines.\n5. Measurable residual disease based on imaging findings using Curie scoring or MIBG or PET imaging criteria (1: measurable tumor of at least 10 mm in one dimension on MRI or CT scan with positive uptake on I-123 MIBG scan (\"MIBG avid\") oOR 2): increased FDG uptake on 18F-FDG PET-CT or PET-MRI (\"PET avid\")).\n\n   \\-\n\nExclusion Criteria:\n\n1. Insufficient bone marrow function: Platelet count \\> 50,000\u002FµL, independent of transfusion (no platelet transfusion within one week). Absolute neutrophil count (ANC) maximum of 500 per microliter. Hemoglobin \\> 10 grams per deciliter.\n2. Insufficient liver function: Plasma bilirubin level more than 1.5 times the upper limit of normal (ULN). SGPT (ALT) at least three times the upper limit of normal (a level of 45 units per liter is considered the upper limit of normal).\n3. Insufficient kidney function: Creatinine clearance or estimated radioisotope GFR \\\u003C 70 ml\u002Fmin\u002F1.73m². Plasma creatinine level more than 1.5 times the upper limit of normal based on age\u002Fgender.\n4. Insufficient central nervous system function if seizures are present, entry into the study is not possible and if seizures are not well controlled with anticonvulsant drugs.\n\n3- Insufficient cardiovascular function Shortening fraction \\\u003C 27% by ECHO OR Ejection fraction \\\u003C 50% by ECHO or gated radionuclide study.\n\n4- Insufficient pulmonary function evidence of dyspnea at rest. Exercise intolerance. Chronic need for oxygen and room air pulse oximetry \\\u003C 94% if pulse oximetry evaluation is clinically indicated. Presence of current pleural or pericardial effusion.\n\n5- Inability to tolerate new treatment due to emergency conditions. 6- Elevated catecholamines (more than twice the ULN) or sole involvement of bone marrow (bone marrow positive for NB as the only evaluable disease without confirmatory pathology report).\n\n7- Receiving 0.5 mg\u002Fkg\u002Fday of systemic steroids (equivalent to prednisone) for at least 7 days before enrollment.\n\n8- Receiving CYP3A4 inducers or inhibitors at least 7 days before study enrollment.\n\n9- Diagnosis of any other malignancy alongside the diagnosis of neuroblastoma. 10- Diarrhea \\> Grade 2 (4 to 6 stools per day). 11- Significant illness not covered by exclusion criteria but interfering with the study process or increasing the intensity of treatment with NK cells.\n\n12- Participation in another clinical trial. 13- Severe impairment of major organ functions, such as renal, cardiac, hepatic, neurological, pulmonary, or gastrointestinal toxicity above Grade 2 according to the National Cancer Institute's Common Terminology Criteria for Adverse Events version 5.0 (CTC v5.0).\n\n14- Inability to comply with protocol requirements. 15- Lack of confirmed and signed consent by the patient's guardians. 16- Evidence of HIV disease (Human Immunodeficiency Virus) or positive serology for HIV.","2 Years","16 Years",{"count":110,"type":19},10,[112],"PHASE1","The goal of this clinical trial is to assess safety and efficacy of systemic injection of allogenic NK cells in patients with refractory\u002Frecurrent high-risk neuroblastoma.\n\nIs the injection of allogenic nk cells safe in patients with R\u002FR high-risk neuroblastoma? Is the injection of allogenic nk cells effective in patients with R\u002FR high-risk neuroblastoma? We will compare the NK cell administration group with a control group that receives conventional treatment to determine whether the intervention is safe and effective",[115,25,26],"Neuroblastoma, Recurrent, Refractory",[117,118,119,120,121],"Neuroblastoma","High-Risk Neuroblastoma","Natural Killer Cell","Nk Cell","adrenal gland","2025-04-05",{"date":124,"type":31},"2025-04-09",{"date":126,"type":31},"2024-11-10",{"date":128,"type":19},"2026-11",{"name":130,"class":131},"Marzieh Ebrahimi","INDUSTRY",{"id":133,"slug":134,"hasResults":11,"nctId":135,"briefTitle":136,"officialTitle":137,"acronym":4,"eligibilityCriteria":138,"healthyVolunteers":11,"sex":16,"minAge":139,"maxAge":108,"enrollmentInfo":140,"targetDuration":142,"studyType":83,"phases":4,"briefSummary":143,"conditions":144,"keywords":150,"overallStatus":157,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":159,"startDateStruct":161,"completionDateStruct":163,"leadSponsor":165,"locationsCount":39},"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",{"count":141,"type":19},360,"2 Weeks","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.",[145,146,147,25,148,149],"Quality of Life (QOL)","Patient-Reported Outcome Measures (PROMs)","Pneumonia Childhood","Wilms Tumour","Immune Thrombocytopenic Purpura ( ITP )",[151,60,152,153,154,155,156],"Quality of life","psychometric measurements","Paediatric pneumonia","Paediatric central nervous system solid tumour","paediatric immune thrombocytopenic purpura","spillover effect","NOT_YET_RECRUITING","2025-03-07",{"date":160,"type":31},"2025-03-13",{"date":162,"type":19},"2025-04-01",{"date":164,"type":19},"2027-12-31",{"name":166,"class":38},"RenJi Hospital",{"id":168,"slug":169,"hasResults":11,"nctId":170,"briefTitle":171,"officialTitle":171,"acronym":4,"eligibilityCriteria":172,"healthyVolunteers":11,"sex":16,"minAge":173,"maxAge":174,"enrollmentInfo":175,"targetDuration":4,"studyType":20,"phases":176,"briefSummary":177,"conditions":178,"keywords":180,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":182,"lastUpdatePostDateStruct":183,"startDateStruct":185,"completionDateStruct":187,"leadSponsor":189,"locationsCount":191},"100580377","phase-1-safety-and-efficacy-of-car-t-cell-therapy-for-relapsedrefractory-neuroblastoma-and-desmoplastic-small-round-cell-tumors-a-single-arm-open-label-trial-100580377","NCT06836505","Safety and Efficacy of CAR-T Cell Therapy for Relapsed\u002Frefractory Neuroblastoma and Desmoplastic Small Round Cell Tumors: a Single-arm, Open-label Trial.","Inclusion Criteria:\n\n1. Patients who are diagnosed as relapsed\u002Frefractory neuroblastoma or relapsed\u002Frefractory desmoplastic small round cell tumors;\n2. Age 1-50 years, any gender;\n3. Agree to participate in the trial and sign a written informed consent form;\n4. Expected survival of ≥12 weeks;\n5. Karnofsky performance status (for patients ≥16 years) or Lansky performance status (for patients \\\u003C16 years) (Appendix 1) must be at least 50;\n6. Good function of major organs:\n\n   1. Liver function: ALT ≤ 5 times the upper limit of normal for the corresponding age, and bilirubin ≤ 2.0 mg\u002FdL, except for patients with Gilbert-Meulengracht syndrome. Patients with Gilbert-Meulengracht syndrome who have bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included;\n   2. Renal function: Plasma creatinine ≤ 1.5 times the upper limit of normal, or estimated glomerular filtration rate (eGFR) ≥ 60 mL\u002Fmin\u002F1.73m²;\n   3. Pulmonary function: Oxygen saturation ≥ 95% in room air;\n   4. Cardiac function: Left ventricular ejection fraction (LVEF) ≥ 45%;\n7. Patients using the following medications must meet the following conditions:\n\n   Steroids: Steroid treatment doses must be stopped at least 2 weeks before CAR-T infusion. However, physiological replacement doses of steroids are allowed; Immunosuppressants: Any immunosuppressive drugs must be stopped at least 4 weeks before enrollment; Anti-proliferative treatments other than lymphodepleting chemotherapy within two weeks before infusion; CNS disease prophylaxis must be stopped 1 week prior to CAR-T infusion (e.g., intrathecal methotrexate injection);\n8. Patients of childbearing potential (both male and female) must agree to use reliable contraception methods (hormonal or barrier methods or abstinence) with their partner until at least 12 months after CAR-T cell infusion, and until two consecutive flow cytometry or PCR tests show no CAR-T cells in the body;\n9. If the subject cannot provide suitable T cells for CAR-T preparation, T cells from a healthy donor may be collected for preparation.\n\nExclusion Criteria:\n\n* Patients with any of the following items will not be enrolled in this study:\n\n  1. Patients with increased intracranial pressure or altered consciousness;\n  2. Patients who have received radiation therapy within 2 weeks prior to infusion;\n  3. Patients with active hepatitis B (defined as HBV DNA \\> 500 IU\u002FmL) or hepatitis C (HCV RNA positive);\n  4. HIV-positive patients or patients with a positive syphilis test;\n  5. Patients with uncontrolled acute life-threatening bacterial, viral, or fungal infections (e.g., positive blood cultures within ≤72 hours before infusion);\n  6. Patients with unstable angina and\u002For myocardial infarction within 6 months prior to screening;\n  7. Patients with a history of or concurrent malignancies, except for the following conditions:\n\n     1. Basal cell carcinoma or squamous cell carcinoma that has been adequately treated (sufficient wound healing required before study enrollment);\n     2. Carcinoma in situ of the cervix or breast that has been cured, with no signs of recurrence for at least 3 years before the study;\n     3. Primary malignant tumors that have been completely resected and have been in complete remission for ≥5 years;\n  8. Pregnant or breastfeeding female patients;\n  9. Patients with uncontrolled arrhythmias that have not been managed medically;\n  10. Patients who need oral anticoagulation therapy within 1 week before CAR-T cell infusion;\n  11. Patients with active neuroautoimmune or inflammatory diseases (e.g., Guillain-Barré syndrome, amyotrophic lateral sclerosis);\n  12. Other conditions deemed inappropriate for participation in the clinical study by the investigator.\n\nPatients enrolled in the clinical study must meet the inclusion criteria and not meet the exclusion criteria.","1 Year","50 Years",{"count":110,"type":19},[112,22],"Title: Safety and efficacy of CAR-T cell therapy for relapsed\u002Frefractory neuroblastoma and desmoplastic small round cell tumors: a single-arm, open-label trial.\n\nThe CART used in this study will be provided by Shanghai YaKe Biotechnology Ltd.\n\nAims:\n\n1. To evaluate the safety and efficacy of GD2\u002FB7H3 CAR-T therapy for relapsed\u002Frefractory neuroblastoma, and observe its pharmacokinetic\u002Fpharmacodynamic characteristics and the survival of CAR-T cells in relapsed\u002Frefractory neuroblastoma patients.\n2. To evaluate the safety and efficacy of GD2\u002FB7H3 CAR-T therapy for relapsed\u002Frefractory desmoplastic small round cell tumor, and observe its pharmacokinetic\u002Fpharmacodynamic characteristics and the survival of CAR-T cells in desmoplastic small round cell tumor patients.\n\nPatients: Relapsed\u002Frefractory neuroblastoma; Relapsed\u002Frefractory desmoplastic small round cell tumor.\n\nCAR-T therapy: Lymphodepletion treatment will be performed within 14 days prior to CAR-T cell infusion: intravenous chemotherapy based on fludarabine 25mg\u002Fm² and cyclophosphamide 500mg\u002Fm² for 1 to 3 days. CAR-T cells will then be infused intravenously, with a dosage of 1.00 to 10.00 × 10⁶\u002Fkg of CAR-positive T cells.\n\nResearch period: CAR-T cell infusion will be followed up for one year, or until adverse events resolve, progression occurs, or the patient transitions to other treatments.\n\nOutcome measures:\n\nIncidence of adverse events related to CAR-T therapy, as well as their intensity and duration; Pharmacokinetic\u002Fpharmacodynamic characteristics of CAR-T in patients and the survival of CAR-T cells.\n\nOverall response rate (ORR) after CAR-T cell infusion, including complete response (CR) and partial response (PR); Overall survival (OS), progression-free survival (PFS), event-free survival (EFS), time to progression (TTP), and duration of response (DOR) after CAR-T cell infusion;",[25,179],"Desmoplastic Small Round Cell Tumor (DSRCT)",[181],"CART, Neuroblastoma, Desmoplastic Small Round Cell Tumor","2025-02-25",{"date":184,"type":31},"2025-02-26",{"date":186,"type":31},"2024-12-12",{"date":188,"type":19},"2027-12-12",{"name":190,"class":38},"Sun Yat-Sen University Cancer Center",3]