[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100630795":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":28,"locations":37,"responsibleParty":57,"collaborators":59,"id":62,"slug":63,"hasResults":64,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":68,"eligibilityCriteria":69,"healthyVolunteers":64,"sex":70,"minAge":71,"maxAge":72,"enrollmentInfo":73,"targetDuration":11,"studyType":76,"phases":77,"briefSummary":79,"conditions":80,"keywords":11,"overallStatus":40,"whyStopped":11,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":91},{"fullName":5,"class":6},"University of Florida","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"recurrent\u002Fprogressive Medulloblastoma (rMB)","EXPERIMENTAL",null,[13],"Biological: Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":11},"BIOLOGICAL","Autologous total tumor mRNA and pp65 full length (fl) lysosomal associated membrane protein (LAMP) mRNA loaded DOTAP liposome vaccine administered intravenously (RNA loaded lipid particles, RNA-LPs)","RNA-LP vaccines will be administered intravenous. Three RNA-LP vaccines will be administered every 2 weeks followed by 12 cycles of adjuvant monthly RNA-LP vaccines for a total of 15 vaccines.",[9],[21,25],{"name":22,"affiliation":23,"role":24},"Sabine Mueller, MD, PhD","University of California, San Francisco","STUDY_CHAIR",{"name":26,"affiliation":5,"role":27},"Elias Sayour, MD, PhD","PRINCIPAL_INVESTIGATOR",[29,33],{"name":26,"role":30,"phone":31,"phoneExt":11,"email":32},"CONTACT","352-273-9000","Wells-BTC@ufl.edu",{"name":34,"role":30,"phone":35,"phoneExt":11,"email":36},"Jannerfer An","(415) 476-3831","PNOC020@ucsf.edu",[38],{"facility":39,"status":40,"city":41,"state":42,"zip":43,"country":44,"countryCode":45,"cosmosGeoPoint":46,"geoPoint":51,"contacts":52},"UF Health Shands Children's Hospital","RECRUITING","Gainesville","Florida","32608","United States","US",{"type":47,"coordinates":48},"Point",[49,50],-82.32483,29.65163,{"lat":50,"lon":49},[53,55],{"name":54,"role":30,"phone":31,"phoneExt":11,"email":32},"Marcia Hodik, RN",{"name":56,"role":27,"phone":11,"phoneExt":11,"email":11},"John Ligon, MD",{"type":58,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR",[60],{"name":61,"class":6},"Pediatric Neuro-Oncology Consortium","100630795","phase-1-rna-lipid-particle-rna-lp-vaccines-for-recurrentprogressive-medulloblastoma-mb-100630795",false,"NCT07492316","RNA-lipid Particle (RNA-LP) Vaccines for Recurrent\u002FProgressive Medulloblastoma (MB)","A Phase I\u002FII Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM), and Recurrent\u002FProgressive Medulloblastoma (MB)","PNOC020 rMB","Inclusion Criteria:\n\n* Age \\> 3 and \\\u003C\u002F= 39 years.\n* Histologically confirmed or suspected recurrent\u002Fprogressive MB in first or second relapse.\n* Patients must have received radiation therapy as part of prior therapy.\n* Patient must have been enrolled on a screening consent and have had sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs).\n* Prior Therapy: Patients must have fully recovered from all acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately.\n\n  * XRT\u002FExternal Beam Irradiation, including Protons: ≥ 90 days after local XRT; ≥ 150 days after TBI, craniospinal XRT or if radiation to ≥ 50% of the pelvis.\n  * Other therapeutic clinical trials: ≥ 14 days after last dose of investigational agent, unless otherwise defined above.\n  * Patients must not have received prior exposure to pp65-directed therapy or any RNA-LP therapy.\n* A diagnostic contrast-enhanced MRI of the brain and spine must be performed preoperatively, and diagnostic contrast-enhanced MRI of the area biopsied or resected must be performed postoperatively. Pre-op MRI must be performed within 28 days prior to study enrollment. Post-op MRI must be completed within 7 days after surgery.\n* Performance Score: Karnofsky ≥ 60 for participants \\> 16 years of age and Lansky ≥ 60 for participants \\\u003C 16 years of age (See Appendix A) assessed within 2 weeks prior to enrollment. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.\n* Bone Marrow:\n\n  d. ANC (Absolute neutrophil count) ≥ 1,000\u002Fμl (unsupported) e. Platelets ≥ 100\u002Fμl (unsupported for at least 7 days) f. Hemoglobin \\> 8 g\u002FdL (may be supported)\n* Renal: Creatinine clearance or radioisotope GFR ≥ 70mL\u002Fmin\u002F1.73 m2\n* Hepatic:\n\n  d. Bilirubin ≤ 3 times upper limit of institutional normal for age. e. SGPT (ALT) ≤ 5 times upper limit of institutional normal for age. f. SGOT (AST) ≤ 5 times upper limit of institutional normal for age.\n* Participants who are receiving systemically-administered steroids must be on a stable or decreasing dose for \\>1 week prior to enrollment. The patient steroid dose should be no more than a dexamethasone-equivalent of 2.8 mg\u002Fm2\u002Fday. Corticosteroid physiologic replacement therapy for management of pituitary\u002Fadrenal axis insufficiency and\u002For topical administration (e.g. inhaled or dermatologic) is allowed.\n* Willing to take an antiepileptic medication such as levetiracetam for the duration of RNA-LP vaccinations\n* A legal parent\u002Fguardian or patient must be able to understand and be willing to sign a written informed consent document\n* For women of childbearing potential (WOCBP), negative serum\u002Furine pregnancy test at enrollment\n* WOCBP must be willing to use acceptable contraceptive methods to avoid pregnancy throughout the study and for at least 24 weeks after the last dose of study drug.\n* Males of child-fathering potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 24 weeks following the last dose of study drug.\n* Participants with post-surgical neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.\n* Patients must be enrolled on PNOC COMP prior to enrollment on PNOC020 if PNOC COMP is open to accrual at the enrolling institution.\n\nExclusion Criteria:\n\n* Diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E+\n* Bulky disease, defined as:\n\n  * Tumor with evidence of clinically significant uncal herniation, midline shift, tonsillar herniation, or brainstem infiltration, or that shows significant mass effect in either brain or spine\n  * Tumor with extensive and diffuse multilobular involvement (\\>3 lobes)\n  * Tumor with extracranial disease (with the exception of spinal metastases in Stratum 3)\n* Known HIV, Hepatitis B, or Hepatitis C seropositive.\n* Uncontrolled seizure disorder\n* History of myocarditis\n* Receipt of any live vaccine within 30 days prior to enrollment\n* Known active infection or immunosuppressive disease.\n* Participants with significant renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), pulmonary, hepatic or other organ dysfunction.\n* Severe or unstable concurrent medical conditions.\n* Women must not be pregnant or breast-feeding.\n* Participants who are receiving any other investigational agents or who have been treated on any other therapeutic clinical protocols within 30 days prior to study entry.\n* Participants who are unwilling or unable to receive treatment and undergo follow-up evaluations.","ALL","4 Years","39 Years",{"count":74,"type":75},24,"ESTIMATED","INTERVENTIONAL",[78],"PHASE1","The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in pediatric patients with recurrent\u002Fprogressive Medulloblastoma (MB)",[81],"Recurrent Medulloblastoma","2026-06-26",{"date":84,"type":85},"2026-06-29","ACTUAL",{"date":87,"type":85},"2026-04-24",{"date":89,"type":75},"2031-03-31",{"name":5,"class":6},1]