[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"plexiform-neurofibromas\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:plexiform-neurofibromas":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,45,78],{"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":26,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100250838","development-and-validation-of-patient-reported-outcome-pro-measures-for-individuals-with-neurofibromatosis-1-nf1-and-plexiform-neurofibromas-pnfs-100250838",false,"NCT02544022","Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)","* SUBJECT INCLUSION CRITERIA:\n* Documented NF1 either by NIH clinical criteria or molecularly-proven mutation in the\n\nNF1 gene, PER the Neurofibromatosis Diagnostic Criteria AND \\>=1 plexiform neurofibroma in any location that is either symptomatic or asymptomatic, and is defined by the following:\n\n1. a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches OR a spinal neurofibroma that involves two or more levels with connection between the levels or extending laterally along the nerve OR a skin thickness neurofibroma;\n2. measures \\>=3 cm on longest diameter by visual exam, palpation or 2D MR imaging OR \\>=3 mL by volumetric MR imaging.\n\n   * For phase 1, Age \\>=5 years. (complete)\n   * For phase 2, Age \\>= 8 years\n   * Ability of subject or parent or guardian to understand and the willingness to sign a written informed consent document.\n   * Participants must be able to understand, read, and speak the English language.\n   * For phase 1 focus groups only, patients need to report experiencing pNF related pain recently with a minimum pain level of 3 on the current NRS-11 or report taking prescription medication that reduces pain and experiencing pNF related pain recently with a minimum pain level of 1 on the current NRS-11. (complete)\n   * For phase 2 patients with pain, patients need to report recently experiencing at least a minimal amount of pNF-related pain. Specifically, they will be asked if they recently experienced any pain in a target tumor area and will have to respond yes to be eligible.\n   * For phase 2 patients without pain, patients need to report no recent pNF-related pain. Specifically, they will be asked if they recently experienced any pain in a target tumor area and will have to respond no to be eligible.\n\nPRIMARY CAREGIVER INCLUSION CRITERIA:\n\n* Primary caregiver (i.e. parent,guardian, grandparent) who is \\>= 18 years old of participating subject \\\u003C= 17 years old\n* Participants must be able to understand, read, and speak the English language\n\nEXCLUSION CRITERIA:\n\n* Patients with severe cognitive or behavior impairments who, in the judgment of the investigators, would not be able to cooperate with the study procedures will be excluded.\n* Patients cannot be newly enrolled on a clinical trial to treat their pNF or cannot have started a new pain treatment regimen (e.g., medication, psychosocial therapy, physical therapy, etc.) at the time of enrollment. Specifically, patients will be ineligible if they were enrolled on a MEK inhibitor trial in the past 12 months or began a new pain\n\nmedication or treatment within the past 3 months prior to enrollment on this study.",true,"ALL","5 Years",{"count":19,"type":20},476,"ESTIMATED","OBSERVATIONAL","Background:\n\nPeople with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (pNFs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning.\n\nObjectives:\n\nTo examine and improve questionnaires about daily living for people with NF1 and pNFs.\n\nEligibility:\n\nPeople ages 5 and older with NF1 and a pNF\n\nDesign:\n\nParticipants will be screened with medical history.\n\nThis study will have 2 phases.\n\nPhase 1 participants will talk about existing pain assessment questionnaires and how pNFs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and pNFs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms.\n\nPhase 2 is now complete.\n\nPhase 1 participants may be invited to Phase 2.\n\nPhase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice-in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice-before the treatment change and 1 month later.",[24,25],"Neurofibromatosis 1","Plexiform Neurofibromas",[27,28,29,30,31],"Pain Scale","QOL","Tool Validation","Cognitive Interviews","Natural History","RECRUITING","2026-06-17",{"date":35,"type":36},"2026-06-18","ACTUAL",{"date":38,"type":36},"2015-11-13",{"date":40,"type":20},"2026-12-31",{"name":42,"class":43},"National Cancer Institute (NCI)","NIH",6,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":56,"conditions":57,"keywords":63,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":77},"100555728","neurofibromatosis-type-1-tumor-early-detection-study-100555728","NCT06515860","Neurofibromatosis Type 1 Tumor Early Detection Study","Observational Trial of Liquid Biopsy for Malignant Peripheral Nerve Sheath Tumor (MPNST) Among Participants With Neurofibromatosis Type 1","NF1-TED","Inclusion Criteria:\n\n* 18 years and older (adults only)\n* Neurofibromatosis Type 1 (NF1) diagnosis (2021 Revised Diagnostic Criteria, PMID: 34012067)\n* History of plexiform neurofibroma (PN)\n* Able to read and understand English or Spanish\n* Live in the USA\n\nExclusion Criteria:\n\n* Are children (younger than 18 years old)\n* Do not have NF1\n* Have no evidence of PN\n* Are not able to read and understand English or Spanish","18 Years",{"count":55,"type":20},1000,"The goal of this observational study is to determine if a liquid biopsy (i.e. blood test) is an effective clinical tool for monitoring the development of malignant peripheral nerve sheath tumor (MPNST) among adults (18 years and older) with Neurofibromatosis Type 1 (NF1), compared to the current standard of care. The main questions it aims to answer are:\n\nHow effective is liquid biopsy compared to the current standard of care (clinical surveillance and imaging) for early detection of MPNST development among people with NF1? Can liquid biopsy offer a cost-effective method for early detection of MPNST in people with NF1? Also, can liquid biopsy provide earlier detection that potentially leads to better outcomes? Also, can offering liquid biopsy improve access to care for people experiencing barriers to access (such as minority populations or people in rural areas)?\n\nAt baseline, participants will be asked to:\n\n* Complete surveys to provide their demographic and NF1-related health information.\n* Report whether or not they are experiencing MPNST-related symptoms.\n* Provide blood samples (15 mL blood total between three tubes, which is approximately one tablespoon).\n\nEvery six months during the five-year follow-up period, participants will be asked to:\n\n* Complete additional surveys to report whether or not they are experiencing MPNST-related symptoms and\u002For if they have been diagnosed with a new MPNST.\n* Provide an additional blood sample (10 mL blood total in one tube).\n\nIf diagnosed with an MPNST by their healthcare provider during the follow-up period, participants will be asked to:\n\n* Complete an additional survey regarding their diagnosis and symptoms.\n* Provide an additional blood sample (10 mL blood in one tube).\n* In parallel, the study team will request a sample of tumor tissue from the care provider, if available.",[58,24,59,25,60,61,62],"Neurofibromatosis Type 1","Plexiform Neurofibroma","Malignant Peripheral Nerve Sheath Tumor","Malignant Peripheral Nerve Sheath Tumors","Atypical Neurofibroma",[64,65,66],"Liquid biopsy","Cancer surveillance","Tumor early detection","2026-01-08",{"date":69,"type":36},"2026-01-09",{"date":71,"type":36},"2024-08-07",{"date":73,"type":20},"2030-07",{"name":75,"class":76},"David Miller","OTHER",1,{"id":79,"slug":80,"hasResults":11,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":4,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":16,"minAge":53,"maxAge":85,"enrollmentInfo":86,"targetDuration":4,"studyType":88,"phases":89,"briefSummary":91,"conditions":92,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":77},"100464700","phase-2-hl-085-in-adults-with-neurofibromatosis-type-1-nf1-and-inoperable-plexiform-neurofibromas-100464700","NCT05331105","HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas","A Multi-center, Open-label, Single-arm Phase II Study to Evaluate the Efficacy and Safety of HL-085 in the Treatment of Adult Participants With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas","Inclusion Criteria:\n\n* Age: patients must be ≥18 years of age at the time of study entry.\n* Diagnosis: Patients must have inoperable and symptomatic plexiform neurofibromas(PN), and patients must have NF1 mutation or meet at least 1 of the following NF1 diagnostic criteria:\n\n  ① ≥6 cafe-au-lait macules ;\n\n  ② Axillary freckling or freckling in inguinal regions;\n\n  ③ ≥2 Lisch nodules (iris hamartomas);\n\n  ④ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex);\n\n  ⑤ An optic pathway glioma;\n\n  ⑥ First-degree relative with NF1.\n* Patients must have a measurable lesion, defined as at least 3 cm in length, amenable to MRI for efficacy assessment.\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.\n* Patients are able to understand and voluntarily sign a written informed consent form.\n* Patients must be willing and able to complete study procedures and follow-up examinations.\n\nExclusion Criteria:\n\n* Patients who are unable to undergo MRI scans (prosthesis, prosthesis, braces, etc.) or patients with lesions that cannot be evaluated by MRI.\n* Patients do not have adequate organ function.\n* Patients who are unable to take drugs orally, have difficulty swallowing or anything that may lead to inadequate drug absorption.\n* Prior treatment with MEK 1\u002F2 inhibitors.\n* Patients known to be allergic to the ingredients or analogues of the study drug.\n* Patients with previous or current retinal diseases such as retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), central serous retinopathy (CSR), etc. (except retinopathy caused by research diseases).\n* With infections or other uncontrolled disease.\n* Strong CYP2C9 inhibitors or inducers within 7 days before treatment of the study drug.\n* Patients who received surgery within 4 weeks or radiotherapy within 6 weeks before enrollment.\n* Patients who participated in any other clinical study treatment within 4 weeks before enrollment.\n* Patients treated with anti-NF1 treatment with unresolved chronic toxicity.\n* Clinical judgment by the investigator that the patient should not participate in the study.","80 Years",{"count":87,"type":20},70,"INTERVENTIONAL",[90],"PHASE2","This is a Multi-center, Open-label, Single-arm Phase II Study to Evaluate the Efficacy and Safety of HL-085 in the treatment of Adult Participants with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas(PN)",[24,25],"2023-05-29",{"date":95,"type":36},"2023-05-31",{"date":97,"type":36},"2021-10-18",{"date":99,"type":20},"2028-10-31",{"name":101,"class":102},"Shanghai Kechow Pharma, Inc.","INDUSTRY"]