Actively Recruiting

Phase 1
Age: 4Years +
All Genders
NCT04573140

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

Led by University of Florida · Updated on 2026-04-24

28

Participants Needed

1

Research Sites

393 weeks

Total Duration

On this page

Sponsors

U

University of Florida

Lead Sponsor

P

Pediatric Neuro-Oncology Consortium

Collaborating Sponsor

AI-Summary

What this Trial Is About

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 (Stratum 1) adult patients with newly diagnosed GBM (MGMT low level or unmethylated in adults only) and (Stratum 2) in pediatric patients with newly diagnosed HGG (pHGG). Funding Source - FDA OOPD

CONDITIONS

Official Title

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

Who Can Participate

Age: 4Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 21 years or older with newly diagnosed de novo glioblastoma (WHO Grade IV) that is MGMT low level or unmethylated
  • Tumor must have a supratentorial component
  • Sterile collection of tumor material suitable for RNA extraction and lipid particle loading
  • Residual post-surgical tumor size 3 cm or less on MRI
  • Recovery from surgery and related complications
  • Preoperative MRI within 28 days and postoperative MRI within 7 days before enrollment
  • Karnofsky performance score 60 or higher (ambulatory status considered)
  • Adequate bone marrow function: ANC ≥ 1,000/µl, platelets ≥ 150/µl, hemoglobin > 8 g/dL
  • Adequate kidney function: BUN ≤ 25 mg/dl, creatinine ≤ 1.7 mg/dl
  • Adequate liver function: bilirubin ≤ 2.0 mg/dl, ALT and AST ≤ 5 times upper limits of normal
  • Willingness to take antiepileptic medication during vaccinations
  • Signed informed consent
  • Women of childbearing potential (WOCBP) must have negative pregnancy test and agree to use contraceptives during and 24 weeks after study
  • Males with partners of childbearing potential must agree to use approved contraception during and 24 weeks after study
  • Stable post-surgical neurological deficits for at least 1 week
  • Pediatric participants aged over 3 and up to 25 years with histologically confirmed WHO Grade III or IV malignant glioma
  • Pediatric participants must meet similar criteria for tumor collection, residual tumor size, recovery, MRI timing, and performance scores (Karnofsky ≥ 60 for >16 years, Lansky ≥ 60 for <16 years)
  • Pediatric adequate bone marrow function: ANC ≥ 1,000/µl, platelets ≥ 100/µl, hemoglobin > 8 g/dL (may be supported)
  • Pediatric adequate renal and liver function within defined limits
  • Stable or decreasing steroid dose ≤ dexamethasone 2.8 mg/m2/day if on steroids
  • Legal guardian or patient able to provide informed consent or assent
  • Enrollment on PNOC COMP prior to PNOC020 if applicable
Not Eligible

You will not qualify if you...

  • Prior invasive malignancy within 3 years except non-melanomatous skin cancer or carcinoma in situ
  • MGMT methylated tumors
  • Gliomatosis cerebri
  • Metastases below tentorium, beyond cranial vault, or leptomeningeal involvement
  • Recurrent or multifocal malignant gliomas
  • Residual tumor burden greater than 3 cm post-surgery
  • Known HIV, Hepatitis B or C infection
  • Active infection or immunosuppressive disease
  • Use of corticosteroids above physiologic doses or not weaned within 1 week of vaccination
  • Prior chemotherapy or radiosensitizers except temozolomide during radiation for GBM
  • Prior overlapping radiotherapy to head or neck
  • Severe active comorbidities including unstable angina, heart failure, recent myocardial infarction, infections requiring IV antibiotics, respiratory illness, hepatic insufficiency, AIDS, autoimmune disease needing immunosuppressants, major medical or psychiatric illness
  • Pregnancy or breastfeeding
  • History of brachial neuritis or Guillain-Barré syndrome
  • Participation in other investigational treatments within 30 days
  • Inability or unwillingness to receive treatment or follow-up
  • Pediatric exclusions include diffuse intrinsic pontine glioma, brainstem midline glioma, BRAFV600E+ tumors
  • Bulky disease with significant herniation, mass effect, multilobular involvement (>3 lobes), or extracranial disease
  • Uncontrolled seizures or history of myocarditis
  • Receipt of live vaccine within 30 days
  • Significant organ dysfunction or unstable medical conditions
  • Pregnancy or breastfeeding
  • Recent investigational treatments or inability to comply with study procedures

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

UF Health

Gainesville, Florida, United States, 32610

Actively Recruiting

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Research Team

M

Marcia Hodik

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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