[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"retinoblastoma\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:retinoblastoma":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,17,0,[8,50,126,151,177,206,227,252,272,294,322,350,376,400,421,449,468],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":15,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":29,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":4,"leadSponsor":46,"locationsCount":49},"100141241","comprehensive-omics-analysis-of-pediatric-and-adult-solid-tumors-and-establishment-of-a-repository-for-related-biological-studies-100141241",false,"NCT01109394","Comprehensive Omics Analysis of Pediatric and Adult Solid Tumors and Establishment of a Repository for Related Biological Studies","* SUBJECT INCLUSION CRITERIA:\n\nPediatric or adult subjects with one of the following:\n\n* Diagnosis of any tumor, malignancy, pre-malignant disorder, or suspected premalignant familial syndromes, regardless, of patient age;\n* Biological relatives of any patient with a tumor, malignancy, pre-malignant disorder, or suspected familial pre-malignant syndrome, regardless of patient age or the diagnosis of an adult malignancy or pre-malignant disorder;\n* Healthy Volunteer without history of malignancy nor a family member currently being treated for cancer who are undergoing surgery, treatment or during well visits;\n* Biospecimens can be collected with minimal additional risk to the subject during sampling or procedures required for routine patient care.\n* Human samples, specimens and data collected on IRB approved protocols that are now closed\n* Ability of subject, Legally Authorized Representative (LAR), or parent\u002Flegal guardian of children \\\u003C=18 to understand and be willing to sign an IRB-approved informed consent document that permits the use of the tumor and other samples for genomic-based molecular characterization projects.\n\nInclusion Criteria for Social and Behavioral Outcome Interviews:\n\n* Parent\u002Fcaregiver of a participating pediatric or adult patient who is being treated for, or who has previously been treated for any form of pediatric cancer.\n* Must be able to give consent and sign the informed consent document.\n* Able to understand the English language.\n\nEXCLUSION CRITERIA:\n\nNone",true,"ALL","4 Weeks",{"count":19,"type":20},6035,"ESTIMATED","OBSERVATIONAL","Background:\n\n\\- Laboratory investigators who are studying common childhood cancers are interested in developing a tissue repository to collect and store blood, serum, tissue, urine, or tumors of children who have cancer or adults who have common childhood cancers. To develop this repository, additional samples will be collected from children and adults who have been diagnosed with common childhood cancers such as leukemia and tumors of the central nervous system.\n\nObjectives:\n\n\\- To collect and store blood, serum, tissue, urine, or tumor samples of children who have cancer or adults who have common childhood cancers.\n\nEligibility:\n\n* Individuals who have been diagnosed with a common childhood cancer (e.g., leukemia) regardless of patient age.\n* Children, adolescents, and adults who have been diagnosed with a type of cancer more commonly found in adults.\n\nDesign:\n\n* Extra blood, serum (the liquid part of blood), tissue, urine, or tumor samples will be collected from participants at a time when sampling is required for medical care or as part of a research study.\n* No additional procedures will be performed for the sole purpose of obtaining additional tumor tissue, aside from what is required for clinical care.",[24,25,26,27,28],"Sarcoma","Endocrine Tumors","Neuroblastoma","Retinoblastoma","Renal Cancer",[30,31,32,33,34,35,36,37,24,38,26],"Genomics","Proteomics","Tissue Repository","Omics","Cell Lines","Natural History","Pediatric Cancer","Solid Tumor","Kidney Cancer","RECRUITING","2026-06-23",{"date":42,"type":43},"2026-06-24","ACTUAL",{"date":45,"type":43},"2010-04-21",{"name":47,"class":48},"National Cancer Institute (NCI)","NIH",5,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":57,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":59,"conditions":60,"keywords":105,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":115,"lastUpdatePostDateStruct":116,"startDateStruct":118,"completionDateStruct":120,"leadSponsor":122,"locationsCount":125},"100289631","familial-investigations-of-childhood-cancer-predisposition-100289631","NCT03050268","Familial Investigations of Childhood Cancer Predisposition","SJFAMILY","NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown below, you may enroll regardless of the results of your clinical genetic testing.\n\nDEFINITION OF FAMILIAR CANCER FOR THIS PROTOCOL:\n\nIn this protocol, the definition of \"Familial Cancer\" is met if any of the following is present:\n\n* An individual with a history of cancer diagnosed under 26 years of age who has at least one first, second or third degree relative with a history of cancer diagnosed under 51 years of age; OR\n* An individual who has been diagnosed with more than one cancer, at least one of which was diagnosed under 26 years of age; OR\n* An individual with a clinical or molecular diagnosis of a known cancer predisposition syndrome; OR\n* An individual with a congenital cancer diagnosed before 6 months of age; OR\n* An individual with a rare pediatric cancer or tumor diagnosed before 26 years of age\n\nº Excluding human papilloma virus-associated cervical cancer and non-melanoma skin cancer occurring in adults.\n\nINCLUSION CRITERIA:\n\n* An individual who meets this protocol's definition of \"Familial Cancer,\" as above.\n* Biologic relatives of an individual meeting this protocol's definition of \"Familial Cancer,\" who are either affected or unaffected by cancer.\n\nEXCLUSION CRITERIA:\n\n* An inability or unwillingness of the research participant or his\u002Fher legally authorized representative (LAR) to provide written informed consent.\n* The participant has received allogeneic bone marrow transplantation and has NO pre-transplant germline (cancer-unaffected) DNA available AND is unwilling to provide a skin sample.",{"count":58,"type":20},1500,"NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown in the Eligibility Section, you may enroll regardless of the results of your clinical genetic testing.\n\nWhile it is well recognized that hereditary factors contribute to the development of a subset of human cancers, the cause for many cancers remains unknown. The application of next generation sequencing (NGS) technologies has expanded knowledge in the field of hereditary cancer predisposition. Currently, more than 100 cancer predisposing genes have been identified, and it is now estimated that approximately 10% of all cancer patients have an underlying genetic predisposition.\n\nThe purpose of this protocol is to identify novel cancer predisposing genes and\u002For genetic variants. For this study, the investigators will establish a Data Registry linked to a Repository of biological samples. Health information, blood samples and occasionally leftover tumor samples will be collected from individuals with familial cancer. The investigators will use NGS approaches to find changes in genes that may be important in the development of familial cancer. The information gained from this study may provide new and better ways to diagnose and care for people with hereditary cancer.\n\nPRIMARY OBJECTIVE:\n\n* Establish a registry of families with clustering of cancer in which clinical data are linked to a repository of cryopreserved blood cells, germline DNA, and tumor tissues from the proband and other family members.\n\nSECONDARY OBJECTIVE:\n\n* Identify novel cancer predisposing genes and\u002For genetic variants in families with clustering of cancer for which the underlying genetic basis is unknown.",[61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,80,81,82,83,84,85,86,87,88,89,26,90,91,92,93,94,95,96,97,98,99,27,100,101,102,103,104],"Acute Leukemia","Adenomatous Polyposis","Adrenocortical Carcinoma","AML","BAP1 Tumor Predisposition Syndrome","Carney Complex","Choroid Plexus Carcinoma","Constitutional Mismatch Repair Deficiency Syndrome","Diamond-Blackfan Anemia","DICER1 Syndrome","Dyskeratosis Congenita","Emberger Syndrome","Familial Acute Myeloid Leukemia","Familial Adenomatous Polyposis","Fanconi Anemia","Familial Cancer","Familial Wilms Tumor","Familial Neuroblastoma","GIST","Hereditary Breast and Ovarian Cancer","Hereditary Paraganglioma-Pheochromocytoma Syndrome","Hodgkin Lymphoma","Juvenile Polyposis","Li-Fraumeni Syndrome","Lynch Syndrome","MDS","Melanoma Syndrome","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Neurofibromatosis Type 1","Neurofibromatosis Type II","Nevoid Basal Cell Carcinoma Syndrome","Non Hodgkin Lymphoma","Noonan Syndrome and Other Rasopathy","Overgrowth Syndromes","Pancreatic Cancer","Peutz-Jeghers Syndrome","Pheochromocytoma\u002FParaganglioma","PTEN Hamartoma Tumor Syndrome","Rhabdoid Tumor Predisposition Syndrome","Rhabdomyosarcoma","Rothmund-Thomson Syndrome","Tuberous Sclerosis","Von Hippel-Lindau Disease",[106,107,108,109,110,111,112,113,114],"Familial cancer","Genetic predisposition","Heritable disease","Cancer risk","Genome analysis","Genetic modifiers","Next generation sequencing (NGS)","Genetic counseling","DNA","2026-06-15",{"date":117,"type":43},"2026-06-17",{"date":119,"type":43},"2017-04-06",{"date":121,"type":20},"2037-03-31",{"name":123,"class":124},"St. Jude Children's Research Hospital","OTHER",1,{"id":127,"slug":128,"hasResults":11,"nctId":129,"briefTitle":130,"officialTitle":131,"acronym":4,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":16,"minAge":133,"maxAge":134,"enrollmentInfo":135,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":137,"conditions":138,"keywords":140,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":143,"lastUpdatePostDateStruct":144,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":125},"100327688","determining-whether-multiple-anesthesia-exposures-affect-cognitive-function-for-retinoblastoma-patients-100327688","NCT03546387","Determining Whether Multiple Anesthesia Exposures Affect Cognitive Function for Retinoblastoma Patients","Incidence of Neurocognitive Deficits in Patients After Treatment for Retinoblastoma With Multiple Anesthesia Exposures","Inclusion Criteria:\n\n* Age ≥ 6 and \\\u003C 17 years old\n\nExclusion Criteria:\n\n* Diagnosis of any secondary disease associated with developmental delay\n* History of second cancer diagnosis\n* Legal Blindness\n* Past surgery of any type, with the exception of enucleation for retinoblastoma\n* Past exposure to anesthesia for surgical intervention\n* More than one anesthesia encounter for surgical intervention before diagnosis at MSK\n* Past External Beam Radiation for treatment of retinoblastoma\n* Past systemic chemotherapy\n* Non-native speaker of English; however, children whose parents speak only Spanish will be included, because the tests for parents are available in Spanish","6 Years","17 Years",{"count":136,"type":20},75,"The purpose of this study is to see if there is a difference between the development of the brain (neurodevelopment) in children who have been treated for retinoblastoma with multiple anesthetic exposures, compared with brain development in children who have never had anesthesia.",[139,27],"Pediatric Retinoblastoma",[141,142],"18-126","Memorial Sloan Kettering Cancer Center","2026-06-01",{"date":145,"type":43},"2026-06-03",{"date":147,"type":43},"2018-05-07",{"date":149,"type":20},"2027-05-07",{"name":142,"class":124},{"id":152,"slug":153,"hasResults":11,"nctId":154,"briefTitle":155,"officialTitle":155,"acronym":4,"eligibilityCriteria":156,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":157,"enrollmentInfo":158,"targetDuration":4,"studyType":160,"phases":161,"briefSummary":163,"conditions":164,"keywords":165,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":166,"lastUpdatePostDateStruct":167,"startDateStruct":169,"completionDateStruct":171,"leadSponsor":173,"locationsCount":176},"100568316","phase-2-retinoblastoma-phase-ii-expanded-access-clinical-trial-100568316","NCT06679634","Retinoblastoma Phase II Expanded Access Clinical Trial","Inclusion Criteria:\n\n* Age: Participants must be \\\u003C 8 years of age.\n* Diagnosis and Treatment. Participants must have: Group D or earlier-stage intraocular retinoblastoma in which enucleation is a recommended therapy, OR Active residual or recurrent intraocular retinoblastoma in at least one eye following completion of first- line therapy (focal therapy for IIRC Group A eyes, or systemic or intra-arterial chemotherapy).\n\nIf both eyes require treatment, there will receive independent and individual treatment. Non-study eye will be treated by standard of care, with only focal therapy during the Study Period, if required.\n\n* Must have demonstrated intraocular calcium in the tumor-containing eye by ophthalmic ultrasound or by neuroimaging as part of standard retinoblastoma diagnosis.\n* Study eye must have vision potential, at least light perception vision in the tumor-bearing eye either with pupil response testing or demonstration of avoidance behavior to light presentation in the affected eye, and no clinical features suggestive of high risk of extraocular extension.\n* Performance Level: Lansky ≥ 50 (\\\u003C16 years of age); Karnofsky performance scale of ≥50 (≥16 years of age).\n* Organ Function Requirements:\n\n  1. Adequate Bone Marrow Function defined as:\n\n     * Peripheral absolute neutrophil count (ANC) ≥ 1000\u002Fmm3\n     * Platelet count ≥ 100,000\u002Fmm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 Days prior to enrollment)\n     * Hemoglobin ≥ 8.0 g\u002FdL at baseline (may receive RBC transfusions)\n  2. Adequate Renal Function defined as:\n\n     * Creatinine clearance or radioisotope GFR ≥70ml\u002Fmin\u002F1.73 m2 or\n     * A serum creatinine based on age\u002Fgender as follows:\n\n     Age Maximum Serum Creatinine (mg\u002FdL) Male Female\n\n     1 month to \\\u003C 6 months 0.4 0.4 6 months to \\\u003C 1 year 0.5 0.5\n     1. to \\\u003C 2 years 0.6 0.6\n     2. to \\\u003C 6 years 0.8 0.8\n\n     6 to \\\u003C 7 years 1 1\n\n     The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the CDC.\n  3. Adequate Liver Function defined as:\n\n     * Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age.\n     * SGPT (ALT) ≤ 110 U\u002FL. For the purpose of this study, the ULN for SGPT is 45 U\u002FL.\n     * Serum albumin ≥ 2 g\u002FdL.\n  4. Pregnancy prevention. Females of reproductive potential must agree to the use of highly effective contraception during study participation and for an additional 40 days after the end of Episcleral Topotecan administration.\n  5. Informed consent. All participants and\u002For their parents or legally authorized representatives must have the ability to understand and the willingness to sign a written informed consent. Assent, where appropriate, will also be obtained.\n\nExclusion Criteria:\n\n* Disease status. Participants known to have any of the following are excluded:\n\n  1. tumor involving the optic nerve rim\n  2. clinical or EUA evidence of extraocular extension\n  3. evidence of metastatic retinoblastoma\n  4. existing neuroimaging showing suspicion of, or definitive, optic nerve invasion, trilateral retinoblastoma or extra-ocular extension.\n* Allergy. Participants with reported allergy to topotecan, camptothecin or derivatives thereof are excluded.\n* Concomitant treatment. Participants who have received chemotherapy, other focal retinoblastoma therapy or any other investigational agent within 3 weeks of Episcleral Topotecan placement are not eligible.\n* Uncontrolled intercurrent illness. Participants with known uncontrolled intercurrent illness that, in the investigator's opinion, would put the participant at undue risk or limit compliance with the study requirements, are not eligible.\n* Febrile illness. Participants with clinically significant febrile illness (as determined by the investigator) within one week prior to initiation of protocol therapy are excluded.\n* Pregnancy and lactation. Females of reproductive potential must have a negative serum pregnancy test within 72 hours prior to initiation of protocol therapy. Due to the unknown but potential risk for adverse events (AEs) in nursing infants secondary to treatment of the mother with the study agents, breastfeeding must be discontinued if the mother is treated on study.\n* Compliance. Any condition of diagnosis that could in the opinion of the Principal Investigator or delegate interfere with the participant's ability to comply with the study instruction, might confound the interpretation of the study results, or put the participant at risk.","8 Years",{"count":159,"type":20},30,"INTERVENTIONAL",[162],"PHASE2","In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with retinoblastoma. The goal of the study is to further determine\u002Fassess the safety and efficacy and optimal chemotherapy dose for retinoblastoma.",[27],[27],"2026-05-19",{"date":168,"type":43},"2026-05-22",{"date":170,"type":43},"2025-02-21",{"date":172,"type":20},"2029-12",{"name":174,"class":175},"Targeted Therapy Technologies, LLC","INDUSTRY",2,{"id":178,"slug":179,"hasResults":11,"nctId":180,"briefTitle":181,"officialTitle":181,"acronym":4,"eligibilityCriteria":182,"healthyVolunteers":11,"sex":16,"minAge":183,"maxAge":184,"enrollmentInfo":185,"targetDuration":187,"studyType":21,"phases":4,"briefSummary":188,"conditions":189,"keywords":192,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":196,"lastUpdatePostDateStruct":197,"startDateStruct":199,"completionDateStruct":201,"leadSponsor":203,"locationsCount":205},"100436120","retinoblastoma-patient-clinical-database-and-tissue-repository-100436120","NCT04959097","Retinoblastoma Patient Clinical Database and Tissue Repository","Inclusion Criteria:\n\n* All patients, age 0 days to 18 years, with a diagnosis unilateral or bilateral retinoblastoma (RB) seen at CHLA (Children's Hospital Los Angeles).\n\nExclusion Criteria:\n\n* Patients referred for second opinions only and not treated at CHLA for any reason.","0 Years","18 Years",{"count":186,"type":20},800,"3 Years","Retinoblastoma (RB) is a primary eye cancer that forms in the back of the eye of infants and toddlers. Traditionally, RB is diagnosed without a biopsy; tumor can only be studied once an eye has been surgically removed.\n\nGiven this limitation, we use aqueous humor (AH), the clear fluid in the front of the eye to detect specific markers, or information, that comes from the tumor itself.",[27,190,191],"Retinoblastoma Bilateral","Retinoblastoma Unilateral",[27,193,194,195],"RB","Ocular oncology","Aqueous humor","2026-04-17",{"date":198,"type":43},"2026-04-22",{"date":200,"type":43},"2017-01-01",{"date":202,"type":20},"2030-12-31",{"name":204,"class":124},"Children's Hospital Los Angeles",9,{"id":207,"slug":208,"hasResults":11,"nctId":209,"briefTitle":210,"officialTitle":211,"acronym":4,"eligibilityCriteria":212,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":213,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":215,"conditions":216,"keywords":4,"overallStatus":217,"whyStopped":4,"lastUpdateSubmitDate":218,"lastUpdatePostDateStruct":219,"startDateStruct":221,"completionDateStruct":223,"leadSponsor":225,"locationsCount":4},"100633728","retinoblastoma-consolidation-in-egyptians-100633728","NCT07530458","Retinoblastoma Consolidation in Egyptians","Retinoblastoma Consolidation: A Retrospective Multivariate Analysis In Egyptian Population","Inclusion Criteria:\n\n* Patients receiving secondary consolidation therapy with at least one year of post-treatment follow-up.\n\nExclusion Criteria:\n\n* Incomplete data, extraocular or orbital RB at initial presentation, or follow-up duration under one year.",{"count":214,"type":20},150,"This analysis will evaluate the investigators' institution's specific experience using Ru-106 isotopes, cryotherapy, or transpupillary thermotherapy (TTT) to treat persistent or recurrent RB in patients who previously underwent systemic IVC or IAC. The investigators aim to detail the demographics, clinical indications, success rates-specifically local control and globe salvage-, predictive factors for success, and complications of each modality of treatment within a tertiary eye care setting in Egypt.",[27],"NOT_YET_RECRUITING","2026-04-08",{"date":220,"type":43},"2026-04-15",{"date":222,"type":20},"2026-05-01",{"date":224,"type":20},"2027-06",{"name":226,"class":124},"Assiut University",{"id":228,"slug":229,"hasResults":11,"nctId":230,"briefTitle":231,"officialTitle":232,"acronym":4,"eligibilityCriteria":233,"healthyVolunteers":15,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":234,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":236,"conditions":237,"keywords":240,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":243,"lastUpdatePostDateStruct":244,"startDateStruct":246,"completionDateStruct":248,"leadSponsor":250,"locationsCount":125},"100571816","detailed-phenotypic-and-genotype-study-to-correlate-rb1-mutations-relating-to-primary-ocular-tumors-and-secondary-extra-ocular-metastasis-100571816","NCT06725173","Detailed Phenotypic and Genotype Study to Correlate RB1 Mutations Relating to Primary Ocular Tumors and Secondary Extra-ocular Metastasis.","Genetic Associations of Ocular Cancers","Inclusion Criteria:\n\n* Patients with molecularly proven retinoblastoma due to RB1 or a typical clinical retinoblastoma phenotype with genetic screening pending.\n* Able to give consent\u002Fparent or guardian able to give consent.\n\nExclusion Criteria:\n\n* Patients unable or unwilling to undertake consent or clinical testing.\n* Patients unwilling to donate a saliva or blood sample in order to establish the genetic cause of their condition.",{"count":235,"type":20},100,"The goal of this observational study is undertake a detailed phenotypic and genotypic study of patients with ocular and secondary cancers due to mutations in the RB1 gene. Our research sequencing approach will allow advanced insight to for further detailed genotypic understanding of parent-of-origin for valuable insight into the genotype-phenotype relationship of this cancer syndrome.",[190,191,238,239,27],"Retinoblastoma, Extraocular","Retinoblastoma, Recurrent",[241,242],"retinoblastoma","parent-of-origin","2026-03-17",{"date":245,"type":43},"2026-03-18",{"date":247,"type":20},"2026-03-16",{"date":249,"type":20},"2031-01-01",{"name":251,"class":124},"University of Washington",{"id":253,"slug":254,"hasResults":11,"nctId":255,"briefTitle":256,"officialTitle":256,"acronym":257,"eligibilityCriteria":258,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":184,"enrollmentInfo":259,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":261,"conditions":262,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":263,"lastUpdatePostDateStruct":264,"startDateStruct":266,"completionDateStruct":268,"leadSponsor":269,"locationsCount":271},"100395158","therapeutic-recommendations-for-the-treatment-of-children-with-a-retinoblastoma-100395158","NCT04425434","Therapeutic Recommendations For The Treatment Of Children With A Retinoblastoma","GFARB12019","Inclusion Criteria:\n\n* Unilateral intraocular Retinoblastoma (RB)\n* Unilateral extraocular intraorbital (RB)\n* Bilateral intraocular (RB)\n* bilateral intraocular (RB) on one side and extraocular but intraorbital on the other side.\n\nExclusion Criteria:\n\n* Externalized tumor mass\n* massive extension to optic nerve up to optical channeltumor\n* intracranial extension leptomeninges\n* cerebral parenchyma\n* extension to regional lymph nodes and\u002For remote metastases.\n* cerebrospinal fluid involvement.\n* Trilateral RB\n* Incapacity to followed the whole treatement.",{"count":260,"type":20},3000,"As the survival of children with retinoblastoma in high income countries is higher than 95% including the bilateral forms this study hopes to improve the outcome in low income countries in Africa by improving early diagnosis and early implementation of this protocol of therapeutic recommendations for treatment.",[27],"2026-02-27",{"date":265,"type":43},"2026-03-02",{"date":267,"type":43},"2020-11-01",{"date":202,"type":20},{"name":270,"class":124},"French Africa Pediatric Oncology Group",7,{"id":273,"slug":274,"hasResults":11,"nctId":275,"briefTitle":276,"officialTitle":277,"acronym":278,"eligibilityCriteria":279,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":280,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":282,"conditions":283,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":284,"lastUpdatePostDateStruct":285,"startDateStruct":287,"completionDateStruct":289,"leadSponsor":291,"locationsCount":125},"100306302","can-pretreatment-mri-be-used-to-predict-intra-arterial-chemotherapy-response-in-retinoblastoma-100306302","NCT03267459","Can Pretreatment MRI be Used to Predict Intra-arterial Chemotherapy Response in Retinoblastoma?","Prediction of the Response Using Dynamic Contrast-enhanced Magnetic Resonance Imaging Before Intra-arterial Chemotherapy in Children With Retinoblastoma","PREDIR","Inclusion Criteria:\n\n* grade B to D retinoblastoma\n* prescription of intra arterial chemotherapy\n\nExclusion Criteria:\n\n* none",{"count":281,"type":20},60,"Retinoblastoma is the most frequent intraocular tumor in children and represents 6% of all pediatric cancers before the age of 5 years-old. The outcome is now excellent with 95 to 97% of 5-years survival rate. Conservative treatments are being more and more used, and intra arterial chemotherapy is one of the adjuvant treatments proposed to the children. The treatment is efficient in most cases, but a small proportion of children will have an early progression after treatment. MRI is used for the diagnosis of retinoblastoma. We aim to find prognostic factors using dynamic contrast-enhanced magnetic resonance imaging (DCE-MRI) for early identification of children response to intra arterial chemotherapy.",[27],"2025-12-30",{"date":286,"type":43},"2026-01-05",{"date":288,"type":43},"2018-01-11",{"date":290,"type":20},"2030-07",{"name":292,"class":293},"Fondation Ophtalmologique Adolphe de Rothschild","NETWORK",{"id":295,"slug":296,"hasResults":11,"nctId":297,"briefTitle":298,"officialTitle":299,"acronym":300,"eligibilityCriteria":301,"healthyVolunteers":11,"sex":16,"minAge":302,"maxAge":4,"enrollmentInfo":303,"targetDuration":4,"studyType":160,"phases":305,"briefSummary":307,"conditions":308,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":313,"lastUpdatePostDateStruct":314,"startDateStruct":316,"completionDateStruct":318,"leadSponsor":320,"locationsCount":125},"100489256","phase-1-gpc2-car-t-cells-for-relapsed-or-refractory-neuroblastoma-and-metastatic-retinoblastoma-100489256","NCT05650749","GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma","GPC2","Neuroblastoma Inclusion Criteria:\n\n1. Patients must be ≥ 1 year of age\n2. Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are also eligible.\n3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma:\n\n   1. That is recurrent\u002Frelapsed or refractory\u002Fpersistent according to INRC AND\n   2. For which standard curative measures do not exist or are no longer effective.\n   3. patients at first relapse are eligible as no known curative therapies exist for relapsed high-risk neuroblastoma.\n4. Patients must have evaluable or measurable disease at enrollment.\n5. In addition, patient must have experienced at least one of the following:\n\n   a. New disease site documented on at least one of the following: i. 123I-meta-iodobenzylguanidine (MIBG) or 18F-mFBG (meta-fluorobenzylguanidine) scan; OR ii. CT\u002FMRI; OR iii. FDG or Ga-68 Dotatate PET (in patients known to have MIBG non-avid tumor) and MRI findings consistent with tumor (i.e., bone lesions), OR iv. Biopsy confirmed neuroblastoma for any new or progressing lesion. b. Greater than 20% increase in a least one dimension of soft tissue mass documented by CT\u002FMRI and a minimum absolute increase of 5 mm in longest dimension in existing lesion(s). Previously irradiated lesions may be included.\n\n   c. Bone marrow biopsy shows progressive disease according to the revised INRC d. Stable persistent disease, such that response at the completion of upfront therapy or salvage therapy is less than partial response AND has a biopsy of at least one site showing viable neuroblastoma.\n\n   e. Responding persistent disease, defined as at least a partial response to frontline therapy (i.e., at least a partial response to frontline therapy but still has residual disease by MIBG scan, CT\u002FMRI, or bone marrow aspirations\u002Fbiopsies). Patients in this category are required to have histologic confirmation of viable neuroblastoma from at least one residual site (tumor seen on routine bone marrow morphology is sufficient).\n6. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n7. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN for age.\n8. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n9. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n10. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n11. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n12. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nNeuroblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Patients with actively progressing CNS metastases, including parenchymal or leptomeningeal involvement. (Note: CNS imaging at screening is only required if the there is a clinical indication of suspected CNS metastasis)\n8. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk of uncontrollable CRS and\u002For neurotoxicity.\n9. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n10. Patients who have received any live vaccines within 30 days prior to enrollment.\n11. Patients who are pregnant or nursing (lactating).\n12. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n\nRetinoblastoma Inclusion Criteria:\n\n1. Patient age ≥ 6 months.\n2. Patients must have metastatic retinoblastoma according to International\n\n   Retinoblastoma Staging System (IRSS) risk classification (4) at the time of study enrollment:\n\n   a. Retinoblastoma Cohort 1 (Extra-CNS metastasis) i. Stage IVa disease ii. Extra-CNS disease must be confirmed as retinoblastoma by histology (either at diagnosis or recurrence) iii. Measurable disease: Defined as \\> 1cm2 or biopsy-proven bone marrow disease iv. Prior treatment: Recurrent or refractory disease following treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy b. Retinoblastoma Cohort 2 (CNS disease) i. Stage IVb disease ii. Histologic confirmation is not required iii. CNS disease defined as measurable disease \\>1cm2, non-measurable, or CSF positivity alone c. Prior treatment: i. Stage IVb.1 and IVb.2: Recurrent after treatment with COG ARET0321-like or equivalent regimen as part of upfront or recurrent therapy i. Stage IVb.3: Prior treatment is not required (i.e., eligible at initial diagnosis or recurrence)\n3. Patients must have a Lansky (≤ 16 years) or Karnofsky (\\> 16 years) score of ≥ 60\n4. Patients must have adequate renal function defined as age-adjusted serum creatinine ≤1.5 ULN .\n5. Total bilirubin ≤ 1.5 x ULN (exception: total bilirubin ≤ 3 ULN for patients with Gilbert's Disease)\n6. Aspartate aminotransferase (AST) ≤ 2.5 ULN (exception: AST ≤ 5 x ULN for patients with liver metastases).\n7. Alanine aminotransferase (ALT) ≤ 2.5 ULN (exception: ALT ≤ 5 x ULN for patients with liver metastases).\n8. Patients must have a baseline pulse oximetry of at least 92% on room air. In addition, a DLCO ≥ 60% (corrected for anemia) is required if PFTs are clinically appropriate as determined by the treating investigator.\n9. Left ventricular shortening fraction (LVSF) ≥28% or ejection fraction (LVEF) ≥ 50% confirmed by Echo, or adequate ventricular function documented by a scan or a cardiologist.\n\nRetinoblastoma Exclusion Criteria:\n\n1. Patients with active hepatitis B or active hepatitis C.\n2. Patients with HIV infection.\n3. Patients with uncontrolled active infection.\n4. Patients with primary or acquired immunodeficiency disorder.\n5. Patients with a known hypersensitivity to DMSO.\n6. Concurrent use of systemic steroids or immunosuppression at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy or immunosuppression during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.\n7. Active medical disorder that, in the opinion of the investigator, would substantially increase the risk to the subject.\n8. Patients with congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), unstable angina, serious uncontrolled cardiac arrhythmia, a myocardial infarction within 6 months prior to study entry or a history of myocarditis.\n9. Patients who have received any live vaccines within 30 days prior to enrollment.\n10. Patients who are pregnant or nursing (lactating).\n11. Patients who have a life expectancy \\\u003C 6 months at time of consent.\n12. Retinoblastoma Cohort 1 (Extra-CNS disease):\n\n    1. Concurrent CNS disease (they may be eligible for Retinoblastoma Cohort 2)\n    2. Stage III disease (orbital or lymph node regional extension without other hematogenous metastases).\n13. Retinoblastoma Cohort 2 (CNS disease):\n\n    1. \"Bulky\" disease (\\>5 cm in diameter) within or compressing the brainstem or thalamus. Note: Tumors touching the brainstem\u002Fthalamus without evidence of compression and\u002For tumors in other CNS locations do not have a maximal size criterion.\n    2. If evidence of clinically significant increased intracranial pressure at time of relapse, patient must demonstrate improvement by time of enrollment.","1 Year",{"count":304,"type":20},45,[306],"PHASE1","This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma or retinoblastoma.",[309,310,311,27,312],"Refractory Neuroblastoma","Relapsed Neuroblastoma","High-risk Neuroblastoma","Metastatic Retinoblastoma","2025-12-24",{"date":315,"type":43},"2025-12-29",{"date":317,"type":43},"2023-05-23",{"date":319,"type":20},"2030-01-30",{"name":321,"class":124},"Stephan Grupp MD PhD",{"id":323,"slug":324,"hasResults":11,"nctId":325,"briefTitle":326,"officialTitle":327,"acronym":328,"eligibilityCriteria":329,"healthyVolunteers":11,"sex":16,"minAge":330,"maxAge":133,"enrollmentInfo":331,"targetDuration":4,"studyType":160,"phases":333,"briefSummary":335,"conditions":336,"keywords":337,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":340,"lastUpdatePostDateStruct":341,"startDateStruct":343,"completionDateStruct":345,"leadSponsor":347,"locationsCount":349},"100414807","phase-2-ocular-conservative-treatment-for-retinoblastoma--efficacy-of-the-new-management-strategies-and-visual-outcome-100414807","NCT04681417","Ocular Conservative Treatment for Retinoblastoma : Efficacy of the New Management Strategies and Visual Outcome","Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018","RETINO 2018","Inclusion Criteria:\n\nOverall study inclusion criteria:\n\n1. Newly diagnosed retinoblastoma (RB).\n2. Retinoblastoma with at least one eye eligible for conservative management.\n3. Patients likely to be compliant with the study requirements and visits, including late follow-up.\n4. Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer.\n5. Patients with no contraindication to the proposed treatments.\n6. Informed consent signed by parents or legal representative.\n7. French Social Security System coverage.\n\nStudy 1 inclusion criteria:\n\n8.1. Children aged from 6 months to 6 years. 9. Retinoblastoma in at least one eye, deemed manageable with IAC in one side and without IV chemotherapy:\n\n1. Unilateral Retinoblastoma classified as group B, C (if vitreous seeds \\\u003C 3 mm), D or E with no invasion of the anterior segment, and without massive tumor of more than 2\u002F3 of the eye, eligible for conservative management, or\n2. Bilateral Retinoblastoma but with very unbalanced lesions between the two eyes: one eye group D or E without invasion of the anterior segment or massive tumor of more than 2\u002F3 of the eye, eligible for IAC, and the other eye eligible for local treatment only (without IAC).\n\nStudy 2 inclusion criteria:\n\n8.2. Retinoblastoma eligible for conservative management although not manageable with IAC:\n\n1. Unilateral Retinoblastoma in children below 6 months of age, classified as Group A, B, C or D, with or without vitreous seeding, compatible with conservative management, or\n2. Bilateral Retinoblastoma classified as group A, B, C D, or E without invasion of the anterior segment, and\u002For massive tumors of more than 2\u002F3 of the eye and eligible for conservative management.\n\nExclusion Criteria:\n\nOverall study non-inclusion criteria:\n\n1. RB not eligible for conservative management :\n\n   1. Extra-ocular extension of the disease, or\n   2. Group E eyes with invasion of the anterior segment, and\u002For massive tumors of more than 2\u002F3 of the eye.\n2. Patient older than 6 years of age.\n3. Patients with another associated disease contra indicating systemic chemotherapy.\n4. Previously treated retinoblastoma by chemotherapy.\n5. Patients already treated for another malignant disease.\n6. Patient with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.\n7. Patients whose parents have not accepted the treatment regimen after explanation of it.\n8. Contraindication to study drug mentioned in SmPC (Summary of Products Characteristics) of the study drugs.\n9. Inclusion in another experimental anti-cancer drug therapy.\n\n   Study 1 non-inclusion criteria:\n10. Any contraindication or concomitant disease that would preclude the Study 1 treatment procedure and could delay treatment.\n\nThese patients should be eligible for Study 2.","6 Months",{"count":332,"type":20},225,[162,334],"PHASE3","This protocol includes 2 independent studies. Both studies are multicenter studies, evaluating different therapeutic approaches in two different populations of patients:\n\n* Study 1, a randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.\n* Study 2, a minimally invasive interventional study evaluating the results on visual function following reference treatment with intravenous (IV) chemotherapy in association with ophthalmologic or local ophthalmological treatment without IV chemotherapy.",[27],[338,339],"Pediatric","Ocular conservative treatment in retinoblastoma","2025-11-21",{"date":342,"type":43},"2025-11-28",{"date":344,"type":43},"2021-03-25",{"date":346,"type":20},"2036-01-20",{"name":348,"class":124},"Institut Curie",28,{"id":351,"slug":352,"hasResults":11,"nctId":353,"briefTitle":354,"officialTitle":355,"acronym":356,"eligibilityCriteria":357,"healthyVolunteers":15,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":358,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":360,"conditions":361,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":366,"lastUpdatePostDateStruct":367,"startDateStruct":369,"completionDateStruct":371,"leadSponsor":373,"locationsCount":375},"100357342","studying-health-outcomes-after-treatment-in-patients-with-retinoblastoma-100357342","NCT03932786","Studying Health Outcomes After Treatment in Patients With Retinoblastoma","Research Into Visual Endpoints and RB Health Outcomes After Treatment (RIVERBOAT)","RIVERBOAT","* Unilateral or bilateral intraocular retinoblastoma\n* Diagnosis between the ages of 0 - 17.99 years\n* Diagnosis on or after January 1, 2008\n* No exclusions based on primary or secondary treatment modalities\n* Retrospective group patients must be ≥ 6 months post end of treatment at study entry\n\n  * For those already at this timepoint, they are now eligible\n  * For those in treatment, or otherwise not yet at this timepoint, they are eligible once at they are ≥ 6 months post end of treatment\n  * Prospective group patients must not have begun treatment\n* Patients with diminished capacity will not be enrolled.\n* Language: Patients must be able to communicate in English, French, or Spanish\n* Sibling Cohort: One sibling, not affected by retinoblastoma will be enrolled, preference for the sibling closest in age to the RB patient.\n* Regulatory Requirements: All patients and\u002For their parents or legal guardians must sign a written informed consent. All institutional, FDA, and NCI requirements for human studies must be met.",{"count":359,"type":20},900,"This trial studies health outcomes after treatment in patients with retinoblastoma. Gathering health information over time from patients and family members through vision assessments, samples of tissue and saliva, and questionnaires may help doctors learn more about what causes retinoblastoma, identify long-term health outcomes for patients with retinoblastoma, and find out which therapies may be the best for treating retinoblastoma",[27,362,363,364,365],"Cancer Survivor","Biological Sibling","Intraocular Retinoblastoma","Unilateral Retinoblastoma","2025-09-15",{"date":368,"type":43},"2025-09-19",{"date":370,"type":43},"2019-01-24",{"date":372,"type":20},"2031-01",{"name":374,"class":124},"Vanderbilt-Ingram Cancer Center",11,{"id":377,"slug":378,"hasResults":11,"nctId":379,"briefTitle":380,"officialTitle":381,"acronym":4,"eligibilityCriteria":382,"healthyVolunteers":11,"sex":16,"minAge":330,"maxAge":4,"enrollmentInfo":383,"targetDuration":4,"studyType":160,"phases":384,"briefSummary":386,"conditions":387,"keywords":389,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":391,"lastUpdatePostDateStruct":392,"startDateStruct":394,"completionDateStruct":396,"leadSponsor":398,"locationsCount":125},"100548692","early-phase-1-targeting-nudt21-sirna-drugs-for-patients-with-refractory-retinoblastoma-100548692","NCT06424301","Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma","Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma (A Prospective Pilot Study)","Inclusion Criteria:\n\n1. Patients with retinoblastoma with a somatic mutation of the gene RB1 and active tumor in a single eye, or germinal mutation of RB1 with active tumor\u002Fs in an eye and the contralateral eye unaffected, enucleated or without tumor activity. In both cases, relapsed or refractory with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy, in accordance with the availability at his\u002Fher referral site, in whom enucleation is the only recommended treatment under opinion of the medical team treating the case at the originating referral site. But the patient has a strong desire to preserve the eye.\n2. Normal renal function: serum creatinine: \\\u003C 45 μmol\u002FL (0-2 years); \\\u003C 57 μmol\u002FL (3-6 years); \\\u003C 60 μmol\u002FL (7-10 years); \\\u003C 80 μmol\u002FL (11-13 years).\n3. Normal Hepatic function: serum ALT: \\\u003C 0,52 μkat\u002FL (de 9 months -12 years); serum AST: 61-80 g\u002FL (8 months - 5 years); 63-83 g\u002FL (5-9 years); 63-82 g\u002FL (9-12 years).\n4. Age greater than 6 months at the time of inclusion in the study.\n5. Sign the informed consent form and be willing to follow up at the specified time.\n\nExclusion Criteria:\n\n1. Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement.\n2. Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment.\n3. Active Infections.\n4. Other chronic or active acute diseases that under the criterion of the researcher were an exclusion criterion.\n5. History of having received attenuated or live vaccines in the 30 days prior to inclusion in the study.\n6. Any cause of Immunosuppression.\n7. Trilateral Retinoblastoma.\n8. Extraocular spread.\n9. History of having received treatment for retinoblastoma with chemotherapy or radiation therapy by any means within 30 days prior to inclusion in the study.\n10. Patients who can not complete the study procedures.",{"count":176,"type":20},[385],"EARLY_PHASE1","Retinoblastoma (RB) is the most common intraocular malignancy in children, accounting for approximately 11% of all cancers diagnosed in children under the age of one. Although its incidence is relatively low-about 1 in 15,000 to 20,000 live births-RB has a high risk of intracranial metastasis via the optic nerve, often leading to poor prognosis in advanced cases.\n\nRecent advances in administration routes, such as intravitreal and intra-arterial chemotherapy, have significantly improved eye preservation rates. However, these strategies are limited by cumulative retinal toxicity and drug resistance. In refractory cases, enucleation remains the only definitive treatment to prevent extraocular spread and death.\n\nIn light of these challenges, current research efforts are focused on developing novel targeted therapies that enhance anti-tumor efficacy while minimizing local toxicity. In this context, we introduce a first-in-class siRNA-based drug targeting NUDT21, which promotes tumor regression by modulating the 3'UTR tail of SMC1A, thereby suppressing tumor cell proliferation. Importantly, the siRNA drug selectively targets tumor cells, offering a favorable safety profile compared to conventional chemotherapeutic regimens.\n\nGiven that both the target (NUDT21) and the mode of administration (intraocular siRNA injection) are novel in retinoblastoma treatment, there is an urgent need for early-phase investigator-initiated clinical research. This study is therefore designed to assess the short-term safety and preliminary efficacy of NUDT21 siRNA in patients with refractory retinoblastoma, and to provide an evidence base for future large-scale clinical trials.",[27,388],"Refractory",[241,390],"intravitreal Chemotherapy","2025-06-20",{"date":393,"type":43},"2025-06-26",{"date":395,"type":43},"2024-12-20",{"date":397,"type":20},"2025-09-30",{"name":399,"class":124},"Eye & ENT Hospital of Fudan University",{"id":401,"slug":402,"hasResults":11,"nctId":403,"briefTitle":404,"officialTitle":405,"acronym":4,"eligibilityCriteria":406,"healthyVolunteers":11,"sex":16,"minAge":302,"maxAge":407,"enrollmentInfo":408,"targetDuration":4,"studyType":160,"phases":410,"briefSummary":411,"conditions":412,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":413,"lastUpdatePostDateStruct":414,"startDateStruct":416,"completionDateStruct":418,"leadSponsor":420,"locationsCount":125},"100590836","early-phase-1-high-dose-topotecan-for-retinoblastoma-with-recurrent-or-refractory-vitreous-seed-100590836","NCT06972602","High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed","High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed(A Prospective Non-controlled Single Arm Trial)","Inclusion Criteria:\n\n1. Patients with retinoblastoma with a somatic mutation of the gene RB1 and active tumor in a single eye, or germinal mutation of RB1 with active tumor\u002Fs in an eye and the contralateral eye unaffected, enucleated or without tumor activity. In both cases, relapsed or refractory vitreous seeding with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy, in accordance with the availability at his\u002Fher referral site, in whom enucleation is the only recommended treatment under opinion of the medical team treating the case at the originating referral site. But the patient has a strong desire to preserve the eye.\n2. Normal renal function: serum creatinine: \\\u003C 45 μmol\u002FL (0-2 years); \\\u003C 57 μmol\u002FL (3-6 years); \\\u003C 60 μmol\u002FL (7-10 years); \\\u003C 80 μmol\u002FL (11-13 years).\n3. Normal Hepatic function: serum ALT: \\\u003C 0,52 μkat\u002FL (de 9 months -12 years); serum AST: 61-80 g\u002FL (8 months-5 years); 63-83 g\u002FL (5-9 years); 63-82 g\u002FL (9-12 years).\n4. Adequate marrow reserve manifested in an absolute neutrophil count \\> 1000 \u002F mm3, platelets \\> 100,000 \u002F mm3 and hemoglobin\\> 8 g \u002F dl, without transfusional or cytokine support at least one month prior to study entry.\n5. Age greater than 1 year and less than 12 years at the time of inclusion in the study.\n6. Sign the informed consent form and be willing to follow up at the specified time.\n\nExclusion Criteria:\n\n1. Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement.\n2. Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment.\n3. Active Infections.\n4. Other chronic or active acute diseases that under the criterion of the researcher were an exclusion criterion.\n5. History of having received attenuated or live vaccines in the 30 days prior to inclusion in the study.\n6. Any cause of Immunosuppression.\n7. Trilateral Retinoblastoma.\n8. Extraocular spread.\n9. History of having received treatment for retinoblastoma with chemotherapy or radiation therapy by any means within 30 days prior to inclusion in the study.\n10. Patients who can not complete the study procedures for reasons psychologically or socially","12 Years",{"count":409,"type":20},10,[385],"Retinoblastoma is the most common intraocular malignancy in infancy and childhood,with an estimated 8000 new cases globally each year.The major cause of failure in the management of retinoblastoma remains the persistence or recurrence of resistant vitreous seeding.Currently,with the emergence of new administration routes, intravitreal chemotherapy has been used for vitreous seeds and the rate of eye preservation has been effectively improved. However, the use of high doses of chemotherapeutic agents may lead to visual impairments due to long term retinal toxicity and some tumors recur or become resistant to chemotherapeutic agents after treatment. In such cases, ocular resection is the only option to prevent extraocular metastasis and death.\n\nTherefore, studies on retinoblastoma are currently focused on finding new targeted therapies at appropriate doses to increase anti-tumor activity and reduce side effects. In this study, Topotecan at a dosage of 100μg will be used to treat patients with refractory or recurrent retinoblastoma.\n\nOn one hand, topotecan, as a topoisomerase I inhibitor, prevents the reconnection of broken single stranded DNA, causing irreversible DNA damage. On the other hand, topotecan upregulates PTEN protein to restore its inhibitory effect on the PI3K\u002FAKT signaling pathway, thereby jointly promoting tumor cell apoptosis and weakening cell proliferation activity.Topotecan at a dosage of 100μg has been proven safe in animal experiments, and there have been a few retrospective case reports on its application in retinoblastoma, but relevant prospective clinical studies are still lacking.\n\nBased on the above background, this study will explore the feasibility and effectiveness of intravitreal injection of Topotecan at a dosage of 100μg in patients with refractory or recurrent retinoblastoma through a prospective study,while evaluating immune response and visual preservation.",[27,239],"2025-05-07",{"date":415,"type":43},"2025-05-15",{"date":417,"type":43},"2025-04-01",{"date":419,"type":20},"2027-12-31",{"name":399,"class":124},{"id":422,"slug":423,"hasResults":11,"nctId":424,"briefTitle":425,"officialTitle":426,"acronym":427,"eligibilityCriteria":428,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":429,"targetDuration":431,"studyType":21,"phases":4,"briefSummary":432,"conditions":433,"keywords":435,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":440,"lastUpdatePostDateStruct":441,"startDateStruct":443,"completionDateStruct":445,"leadSponsor":447,"locationsCount":125},"100544334","trilateral-retinoblastoma-incidence-and-outcomes-100544334","NCT06367530","Trilateral Retinoblastoma: Incidence and Outcomes","Global Survey of Trilateral Retinoblastoma: Incidence and Outcomes","GS-TRIO","Inclusion Criteria:\n\n* patients diagnosed with retinoblastoma\n\nExclusion Criteria:\n\n* none",{"count":430,"type":20},4351,"5 Years","Patients with heritable retinoblastoma are at risk to develop an intracranial brain tumor, which is often fatal. The investigators intend to look at the incidence and survival of trilateral retinoblastoma (which is a brain tumor that can either be located in the pineal gland or elsewhere in the brain) in retinoblastoma patients globally. All retinoblastoma patients from participating centers will be included. The investigators hypothesize that the apparent incidence of trilateral retinoblastoma (especially the usually later diagnosed pineal trilateral retinoblastoma) in low-income countries will be lower because of low chances of surviving the ocular tumors at about 50% and also because of possible under-diagnosis. Therefore, as retinoblastoma care improves in low-income countries the incidence of (pineal) trilateral retinoblastoma might go up. Knowledge about incidence and survival can help improve health practices in parts of the world where this might be needed. Therefore this global study firstly aims 1) to evaluate survival after trilateral retinoblastoma and factors influencing survival and 2) to evaluate incidence of trilateral retinoblastoma by country income level. The study will run from 2024 through 2027.",[434,27],"Trilateral Retinoblastoma",[436,437,241,438,439],"survival","trilateral retinoblastoma","incidence","global","2024-04-11",{"date":442,"type":43},"2024-04-16",{"date":444,"type":43},"2024-01-01",{"date":446,"type":20},"2029-12-31",{"name":448,"class":124},"Amsterdam UMC, location VUmc",{"id":450,"slug":451,"hasResults":11,"nctId":452,"briefTitle":453,"officialTitle":454,"acronym":4,"eligibilityCriteria":455,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":456,"targetDuration":4,"studyType":160,"phases":458,"briefSummary":459,"conditions":460,"keywords":4,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":461,"lastUpdatePostDateStruct":462,"startDateStruct":464,"completionDateStruct":465,"leadSponsor":466,"locationsCount":125},"100445405","phase-3-adjuvant-chemotherapy-for-high-risk-postenucleation-retinoblastoma-100445405","NCT05080010","Adjuvant Chemotherapy for High-risk Postenucleation Retinoblastoma","Three Cycles of Adjuvant Chemotherapy for the Patients With High-risk Retinoblastoma After Enucleation: Prospective Study","Inclusion Criteria:\n\n* Ability to provide written informed consent and comply with study assessments for the full duration of the study.\n* Definite pathology signs of retinoblastoma, Stage I base on International Retinoblastoma Staging System.\n* Received enucleation in the study eye.\n* Monocular retinoblastoma.\n\nExclusion Criteria:\n\n* Any previous disease in the study eye.\n* Previous participation in any studies of investigational drugs within 1 month preceding Day 0 (excluding vitamins and minerals).\n* History of chemical intervention for retinoblastoma in the study eye.",{"count":457,"type":20},500,[334],"The purpose of this study is to determine the effect of 3 cycles of chemotherapy(CEV) in the treatment of Stage I enucleated retinoblastoma.",[27],"2021-10-02",{"date":463,"type":43},"2021-10-15",{"date":267,"type":43},{"date":202,"type":20},{"name":467,"class":124},"Sun Yat-sen University",{"id":469,"slug":470,"hasResults":11,"nctId":471,"briefTitle":472,"officialTitle":473,"acronym":4,"eligibilityCriteria":474,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":475,"targetDuration":4,"studyType":160,"phases":477,"briefSummary":478,"conditions":479,"keywords":481,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":484,"lastUpdatePostDateStruct":485,"startDateStruct":487,"completionDateStruct":489,"leadSponsor":491,"locationsCount":125},"100423832","phase-3-topotecan-and-melphalan-for-retinoblastoma-100423832","NCT04799002","Topotecan and Melphalan for Retinoblastoma","Choice of Topotecan or Melphalan in Retinoblastoma Patients","Inclusion Criteria:\n\n* diagnosed as retinoblastoma\n* receiving eyeball-sparing treatment\n\nExclusion Criteria:\n\n* disease progression during follow-up",{"count":476,"type":20},50,[334],"The purpose of this study is to evaluate topotecan and melphalan for retinoblastoma patients.",[27,480],"Chemotherapy Effect",[27,482,483],"topotecan","Melphalan","2021-03-12",{"date":486,"type":43},"2021-03-16",{"date":488,"type":43},"2021-03-11",{"date":490,"type":20},"2027-12-30",{"name":467,"class":124}]