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RNA-LP Vaccines for Recurrent Medulloblastoma

This study is testing an experimental treatment called RNA-LP vaccines for children and young adults (ages 4-39) with medulloblastoma (a type of brain tumor) that has come back or is getting worse. The RNA-LP vaccines are made using your own tumor cells and a protein called pp65, delivered in tiny fat particles (lipid particles). This treatment is a type of immunotherapy, which helps your body's immune system fight cancer. The main goals of this first-in-human study are to see if the vaccines can be made successfully, if they are safe, and to find the highest dose that can be given without causing too many side effects. You must have had radiation therapy before and have tumor material collected for vaccine creation. The current recruitment status is unclear.

Study design
This is a Phase 1 study, meaning it's the first time this treatment is being tested in humans. It will involve about 24 participants and will increase the dose of the vaccine to find the safest and most effective amount.
What's involved
You would first have tumor material collected. After surgery and other treatments like radiation, you would receive three RNA-LP vaccines every two weeks, followed by 12 monthly vaccines, for a total of 15 vaccines over about 14 months.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed until death due to any cause.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07492316

RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB)

Recruiting
PHASE1Ages 4–39InterventionalTreatment
University of Florida
~24 participants
Updated 2026-06-29 on ClinicalTrials.gov
What's tested: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)

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Manufacturing feasibility
Measured over from the date of surgery until administration of third vaccine, up to 15 weeks
+2 more outcomes measured

Conditions

Recurrent Medulloblastoma

Explore related trials

Medulloblastoma

NCT07492316

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • UF Health Shands Children's Hospital

    Gainesville, Floridastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Sabine Mueller, MD, PhD · STUDY_CHAIR · University of California, San Francisco
  • Elias Sayour, MD, PhD · PRINCIPAL_INVESTIGATOR · University of Florida

Who to contact

Elias Sayour, MD, PhD
[email protected]
352-273-9000
Email the study team

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Eligibility criteria

Inclusion

Age \> 3 and \</= 39 years.
Histologically confirmed or suspected recurrent/progressive MB in first or second relapse.
Patients must have received radiation therapy as part of prior therapy.
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).
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.
XRT/External Beam Irradiation, including Protons: ≥ 90 days after local XRT; ≥ 150 days after TBI, craniospinal XRT or if radiation to ≥ 50% of the pelvis.
Other therapeutic clinical trials: ≥ 14 days after last dose of investigational agent, unless otherwise defined above.
Patients must not have received prior exposure to pp65-directed therapy or any RNA-LP therapy.
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.
Performance Score: Karnofsky ≥ 60 for participants \> 16 years of age and Lansky ≥ 60 for participants \< 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.
Bone Marrow:
Renal: Creatinine clearance or radioisotope GFR ≥ 70mL/min/1.73 m2
Hepatic:
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/m2/day. Corticosteroid physiologic replacement therapy for management of pituitary/adrenal axis insufficiency and/or topical administration (e.g. inhaled or dermatologic) is allowed.
Willing to take an antiepileptic medication such as levetiracetam for the duration of RNA-LP vaccinations
A legal parent/guardian or patient must be able to understand and be willing to sign a written informed consent document
For women of childbearing potential (WOCBP), negative serum/urine pregnancy test at enrollment
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.
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.
Participants with post-surgical neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment.
Patients must be enrolled on PNOC COMP prior to enrollment on PNOC020 if PNOC COMP is open to accrual at the enrolling institution.

Exclusion

Diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E+
Bulky disease, defined as:
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
Tumor with extensive and diffuse multilobular involvement (\>3 lobes)
Tumor with extracranial disease (with the exception of spinal metastases in Stratum 3)
Known HIV, Hepatitis B, or Hepatitis C seropositive.
Uncontrolled seizure disorder
History of myocarditis
Receipt of any live vaccine within 30 days prior to enrollment
Known active infection or immunosuppressive disease.
Participants with significant renal, cardiac (congestive cardiac failure, myocardial infarction, myocarditis), pulmonary, hepatic or other organ dysfunction.
Severe or unstable concurrent medical conditions.
Women must not be pregnant or breast-feeding.
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.
Participants who are unwilling or unable to receive treatment and undergo follow-up evaluations.

What this trial measures

  • Manufacturing feasibilityfrom the date of surgery until administration of third vaccine, up to 15 weeks

    Manufacturing feasibility will be determined based on the percentage of vaccines that are successfully manufactured in the DLT window during the first three vaccines. If two-thirds of vaccines are successfully manufactured with QA/QC clearance, we will conclude that RNA-LPs can be successfully manufactured.

  • Safety of RNA-LP vaccineFirst vaccine through 14 days after administration of the 3rd vaccine

    Number of patients with DLTs at MTD. DLTs will be monitored for two weeks after the 3rd vaccine before continuing with the next dose escalation. If there are no DLTs, only patients receiving at least 3 vaccines without toxicity will be considered as safe for MTD assessment. Patients receiving less than 3 vaccines will be replaced for safety MTD assessments. Toxicity encountered before the 3rd vaccine will be considered a DLT. If 2 or more DLTs are observed at any dose level, the dose level is determined to be unsafe, and no additional patients will be treated at that level and there will be no escalation beyond that level.

  • Determination of Maximum Tolerated Doseup to 60 months

    A traditional 3+3 phase 1 design will be employed during this study, where dose escalations are planned in groups of three patients. No intra-patient escalation will be allowed, and dose escalation will not be considered until toxicity information is available from at least 3 evaluable patients at the current dose level.