[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neurofibromatosis-type-ii\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neurofibromatosis-type-ii":54},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,92],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":69,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":91},"100289631","familial-investigations-of-childhood-cancer-predisposition-100289631",false,"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.","ALL",{"count":18,"type":19},1500,"ESTIMATED","OBSERVATIONAL","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.",[23,24,25,26,27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51,52,53,54,55,56,57,58,59,60,61,62,63,64,65,66,67,68],"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","Neuroblastoma","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","Retinoblastoma","Rhabdoid Tumor Predisposition Syndrome","Rhabdomyosarcoma","Rothmund-Thomson Syndrome","Tuberous Sclerosis","Von Hippel-Lindau Disease",[70,71,72,73,74,75,76,77,78],"Familial cancer","Genetic predisposition","Heritable disease","Cancer risk","Genome analysis","Genetic modifiers","Next generation sequencing (NGS)","Genetic counseling","DNA","RECRUITING","2026-06-15",{"date":82,"type":83},"2026-06-17","ACTUAL",{"date":85,"type":83},"2017-04-06",{"date":87,"type":19},"2037-03-31",{"name":89,"class":90},"St. Jude Children's Research Hospital","OTHER",1,{"id":93,"slug":94,"hasResults":11,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":16,"minAge":99,"maxAge":4,"enrollmentInfo":100,"targetDuration":4,"studyType":102,"phases":103,"briefSummary":106,"conditions":107,"keywords":4,"overallStatus":109,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":4},"100603069","phase-1-study-to-determine-optimal-dose-evaluate-the-efficacy-and-safety-of-prg-n-01-in-patients-with-neurofibromatosis-type-ii-100603069","NCT07131722","Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II","An Open-Label, Dose-Finding, Phase 1\u002F2a Study to Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II","Inclusion Criteria:\n\n1. Male or female subjects aged 18 years or older at screening\n2. Subjects diagnosed with NF2 clinically or genetically, according to the I-NF-DC 2022 updated NF2 diagnostic criteria\n3. Subjects with tumors due to NF2 who require treatment if they meet one of more of the criteria below but cannot undergo surgical treatment due to high risk of side effects from surgery (e.g., damage to nerve function).\n\n(1) Subjects with progressive tumors (VS, non-VS, meningiomas, ependymomas) confirmed on MRI within 36 months prior to screening (2) Subjects with clinical symptoms (decreased function of affected nerves, such as hearing loss, uncontrolled pain, shortness of breath, difficulty swallowing, decreased motor function, and decreased gait) as judged by the investigator 4) Subjects with ECOG performance status 0 - 1 or Karnofsky performance status 70 or higher 5) Subjects who have appropriate hematological, liver, renal, and blood coagulation functions confirmed based on the following criteria at screening: 6) Subjects who have appropriate cardiac and pulmonary functions confirmed based on the following criteria at screening: 7) Subjects who agree to use sunscreen during the clinical study period. 8) Subjects (or the subject's legal representative) who voluntarily consent and provide written informed consent to participate in this clinical study.\n\n9\\) (Only for Phase 2a) Subjects with one or more measurable NF2-related tumors confirmed on MRI at screening\n\nExclusion Criteria:\n\n1\\) Subjects who have the following past or current medical history confirmed during screening:\n\n(1) Malignant tumor requiring treatment (chemotherapy or radiotherapy) or with disease progression within 2 years prior to screening (2) The following heart-related history\n\n* Uncontrolled hypertension at screening (DBP ≥100 mmHg or SBP ≥160 mmHg despite treatment)\n* Acute coronary syndrome (ACS) within 24 weeks of baseline, clinically significant arrhythmia, cardiomyopathy, unstable angina, NYHA II-IV heart failure, or severe valvular heart disease\n\n  1. Interstitial lung disease or pulmonary fibrosis\n  2. Cystitis or urinary obstruction within 12 weeks prior to screening\n  3. Blood coagulation disorder\n  4. Severe or active infectious disease requiring antibiotics, antivirals, etc. within 4 weeks prior to screening\n  5. Gastrointestinal disease that currently makes oral administration difficult or may affect absorption (e.g., celiac disease, Crohn's disease, intestinal resection)\n  6. Other diseases that are sufficient to affect the clinical study results at the investigator's discretion.\n\n  2\\) Subjects who have confirmed or need the following drug treatments:\n  1. Chemotherapy, immunotherapy, or myelosuppressive chemotherapy within 4 weeks prior to screening (but, within 6 weeks prior to screening for nitrosourea agents or mitomycin C)\n  2. Monoclonal antibody therapy within 12 weeks prior to screening (but, allowing if more than 3 half-lives have elapsed); stem cell transplantation (but, allowing if there is no evidence of active graft-versus-host disease and more than 12 weeks have elapsed since transplantation)\n  3. Other investigational drugs or devices within 4 weeks prior to screening\n  4. Corticosteroids such as prednisone and prednisolone within 1 week prior to screening (but, allowing if treated with low doses)\n  5. Substrate of strong inhibitor, inducer, or transporter (OCT2, MATE1, MATE2-K) of CYP enzymes (CYP2C8, CYP3A) (but, allowing only if more than 5 times the half-life from baseline has elapsed) 3) Subjects who performed a major surgery within 4 weeks prior to screening (but, allowing minor surgical procedures such as catheter replacement therapy and local biopsy).\n\n  4\\) Subjects who received radiation therapy for the purpose of treating tumors due to NF2 within 24 weeks prior to screening or who require total body irradiation during the clinical study period.\n\n  5\\) Subjects with prosthetics or orthopedic devices that may interfere with the volumetric analysis of target lesions via MRI.\n\n  6\\) Subjects with a known severe hypersensitivity to PRG-N-01 or a concomitant medication or the ingredients or a history of allergic reactions to compounds of similar chemical or biological composition.\n\n  7\\) Female subjects who are pregnant or breastfeeding 8) Women of childbearing potential or men who are unwilling to use an appropriate method of contraception from the date of written consent to 12 weeks after the last dose of PRG-N-01.\n\n  9\\) Other subjects who are deemed unsuitable for participation in this clinical study at the investigator's discretion","18 Years",{"count":101,"type":19},25,"INTERVENTIONAL",[104,105],"PHASE1","PHASE2","The goal of this clinical trial is to learn if Trineumin(Code name:PRG-N-01) works to treat Neurofibromatosis Type II(NF2) in adults. It will also learn about the safety and tolerability and toxicity of PRG-N-01. The main questions it aims to answer are:\n\n* What dose was determined as the Maximum Tolerated Dose (MTD) of Trineumin?\n* What dose was explored as the optimal effective dose of Trineumin based on radiographic response?\n* Does Trineumin reduce tumor size or improve participants' quality of life, including hearing function?\n* What medical problems do participants have when taking Trineumin?\n\nParticipants will:\n\n* Take Trineumin every day for 96 weeks\n* Visit the clinic once 1, 4, 8, 12, 18week and every 12 weeks and for checkups and tests",[54,108],"NF2","NOT_YET_RECRUITING","2026-01-21",{"date":112,"type":83},"2026-01-23",{"date":114,"type":19},"2026-07",{"date":116,"type":19},"2028-07",{"name":118,"class":119},"PRG Science & Technology Co., Ltd.","INDUSTRY"]