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RNA-LP Vaccines for Newly Diagnosed High-Grade Gliomas

This study is testing an experimental treatment called RNA-lipid particle (RNA-LP) vaccines for adults with newly diagnosed glioblastoma (GBM) and children with newly diagnosed high-grade glioma (HGG). The RNA-LP vaccines are given through an IV. Researchers want to see if the vaccine can be made successfully, if it's safe, and what the highest safe dose is. You might be able to join if you are an adult (21 years or older) with newly diagnosed GBM (WHO Grade IV) that has a specific genetic marker (MGMT low level or unmethylated), or a child (4 years or older) with newly diagnosed HGG. The study plans to enroll 28 participants. The current status of the study is unclear.

Study design
This is a Phase 1 study, the first time this treatment is being tested in humans. It will involve increasing doses to find the highest safe dose.
What's involved
You would have surgery to collect tumor material, followed by standard radiation and chemotherapy. Then, you would receive three RNA-LP vaccines every two weeks, followed by 12 monthly vaccines, for a total of 15 vaccines.
Compensation
Not stated in the trial record.
Follow-up
The study will monitor your safety for at least 14 days after the third vaccine and determine the maximum tolerated dose for up to 30 months.

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

NCT04573140

A Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM)

Recruiting
PHASE1Ages 4+InterventionalTreatment
University of Florida
~28 participants
Updated 2026-06-25 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
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Manufacturing feasibility
Measured over from the date of surgery until adminstration of third vaccine, up to 20 weeks
+2 more outcomes measured

Conditions

Adult Glioblastoma
High Grade Glioma
WHO Grade III or IV Malignant Glioma

Explore related trials

GlioblastomaMalignant GliomaMGMT Gene

Where it's being run

2 sites across 2 states
District of Columbia1
Florida1

Study leadership

  • Elias Sayour, MD, PhD · STUDY_CHAIR · University of Florida

Who to contact

Marcia Hodik
[email protected]
352-273-5519
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Exclusion

Prior invasive malignancy (except for non-melanomatous skin cancer) unless disease free for ≥ 3 years. (For example, carcinoma in situ of the breast, oral cavity, and cervix are all permissible.)
MGMT Methylated tumors
Gliomatosis Cerebri
Metastases detected below the tentorium or beyond the cranial vault and leptomeningeal involvement.
Recurrent or multifocal malignant gliomas.
Metastatic or leptomeningeal disease
Residual post-surgical disease burden \> 3 cm as defined by longest perpendicular diameter on MRI.
Known HIV, Hepatitis B, or Hepatitis C seropositive.
Known active infection or immunosuppressive disease.
Participants who require corticosteroids above physiologic doses or not weaned to physiologic dosing within 1 week of scheduled vaccination.
Prior chemotherapy or radiosensitizers (including Gliadel wafers) for cancers of the head and neck region, other than TMZ prescribed during radiation for GBM (prior chemotherapy for a different cancer is allowable).
Prior radiotherapy to the head or neck, resulting in overlap of radiation fields. Radiosurgery is not permitted.
Severe, active co-morbidity, defined as follows:
Unstable angina and/or congestive heart failure requiring hospitalization.
Unstable cardiac arrhythmias, abnormalities, or transmural myocardial infarction within the last 6 months.
Acute bacterial or fungal infection requiring intravenous antibiotics at initiation of XRT/TMZ.
Chronic Obstructive Pulmonary Disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy at initiation of XRT/TMZ.
Hepatic insufficiency resulting in clinical jaundice and/or coagulation defects.
Acquired Immune Deficiency Syndrome (AIDS) based upon current CDC definition. The need to exclude patients with AIDS from this protocol is necessary because the treatments involved in this protocol may be significantly immunosuppressive.
Patients with autoimmune disease requiring medical management with immunosuppressants.
Major medical illnesses or psychiatric impairments that, in the investigator's opinion, will prevent administration or completion of protocol therapy.
Active connective tissue disorders such as lupus or scleroderma that, in the investigator's opinion, place the patient at high risk for radiation toxicity.
Pregnancy or women of childbearing potential and men who are sexually active and who are unwilling or unable to use an acceptable method of contraception for the entire study period; this exclusion is necessary because the treatment involved in this study may be significantly teratogenic.
Women of childbearing potential must not be pregnant or breast-feeding.
Prior history of brachial neuritis or Guillain-Barré syndrome.
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
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
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 adminstration of third vaccine, up to 20 weeks

    Potentially eligible participants will be enrolled on a screening consent for the sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs). Tumor material will be sent to the University of Florida (UF) where tumor specific RNA-LP vaccines will be manufactured. 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.

    A DLT will be defined as any immunotherapy-related (possible, probable or definite): * Grade ≥ 3 non-hematologic toxicity that does not improve to ≤ Grade 2 within 72 hours; hepatic or renal dysfunction that does not improve to ≤ grade 2 within 7 days * Grade ≥3 cytokine release syndrome that does not improve to ≤ Grade 2 within 72 hours * Grade ≥ 3 encephalopathy as defined by the American Society for Transplantation and Cellular Therapy (ASTCT) Consensus Immune Effector Cell-Associated Encephalopathy criteriaGrade 3 or greater non-neurologic, non-hematologic toxicity * Grade 3 neurologic toxicity that does not improve to Grade II or better within 7 days * Grade 3-4 hematologic toxicity that does not improve to Grade 2 or better within 14 days * Grade 3 autoimmune encephalomyelitis * Grade 4 neurologic toxicity.

  • Determination of Maximum Tolerated Doseup to 30 months

    The Phase I Stratum 1 dose-escalation study will be performed in up to 28 adult participants using a Bayesian optimal interval (BOIN) design with an initial embedded accelerated titration design (ATD) to efficiently identify the maximally tolerated dose (MTD). For the initial ATD, patients will be enrolled and treated in cohorts of size 1 starting at dose level -4 to dose level -1. After the first DLT is observed or if dose level 0 is reached without prior DLT in dose level -4 to -1, the study will expand to a BOIN design with a cohort size of 3 to identify the MTD. For the initial ATD, patients will be enrolled and treated in cohorts of size 1 starting at dose level -4 to dose level -1, and will expand to a cohort size of 3 after the first DLT is observed or at dose level 0. There are 8 dose levels to potentially be assessed including the possibility of 4 dose levels for the ATD and 4 for the BOIN.