Actively Recruiting

Phase 1
Age: 4Years +
All Genders
ID04573140

A Phase I/II Study of RNA-lipid Particle Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas and Adult Glioblastoma

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

28

Participants Needed

1

Research Sites

104 weeks

Total Duration

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Sponsors

U

University of Florida

Lead Sponsor

P

Pediatric Neuro-Oncology Consortium

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating RNA-lipid particle (RNA-LP) vaccines in adults with newly diagnosed glioblastoma (GBM) having low or unmethylated MGMT levels, and in children with newly diagnosed high-grade gliomas (pHGG). This Phase I study aims to assess the manufacturing feasibility, safety, and determine the maximum tolerated dose of these vaccines. The trial includes adult patients with GBM and pediatric patients with HGG, addressing a critical need for novel treatments in these brain cancers. The study involves three parts: surgery, radiation, and immunotherapy. Standard surgery and chemoradiation are performed first, then tumor material is collected for vaccine preparation. The RNA-LP vaccines are given intravenously starting within four weeks after radiation, with three doses every two weeks followed by 12 monthly doses, totaling 15 vaccinations. Participants may continue receiving vaccines for up to 14 months. During the study, participants will undergo regular MRI scans and clinical evaluations to monitor disease progression, initially every three months for one year post-immunotherapy, then every six to twelve months for up to two more years. Researchers will track manufacturing feasibility from surgery until the third vaccine, vaccine safety through two weeks after the third dose, and the maximum tolerated dose over up to 30 months. Follow-up continues until death from any cause to gather long-term information.

CONDITIONS

Brief 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...

  • Adult GBM participants must be 21 years or older with newly diagnosed glioblastoma (WHO Grade IV) that is MGMT low or unmethylated.
  • Tumor must have a supratentorial component with residual post-surgical disease 3 cm or less.
  • Sterile collection of tumor material suitable for RNA vaccine preparation.
  • Must have recovered from surgery and related complications.
  • Pre- and post-operative contrast-enhanced brain MRIs are required.
  • Karnofsky performance score of 60 or higher; ambulatory status considered.
  • Adequate bone marrow, renal, and liver function as specified.
  • Willing to take antiepileptic medication during vaccination.
  • Signed informed consent; pregnancy testing and contraceptive use required for women of childbearing potential.
  • Stable post-surgical neurological deficits for at least 1 week.
  • Pediatric HGG participants aged over 3 and up to 25 years with WHO Grade III or IV glioma.
  • Residual post-surgical disease burden 3 cm or less.
  • Stable or decreasing steroid dose with limits.
  • Informed consent and assent as appropriate.
  • Similar health and laboratory requirements as adults, adjusted for age.
  • Enrolled on PNOC COMP if available before PNOC020 enrollment.
Not Eligible

You will not qualify if you...

  • Prior invasive malignancy unless disease-free for at least 3 years.
  • MGMT methylated tumors.
  • Gliomatosis cerebri or metastatic/leptomeningeal disease.
  • Residual post-surgical tumor greater than 3 cm.
  • Known positive status for HIV, Hepatitis B, or Hepatitis C.
  • Active infections or immunosuppressive diseases.
  • High-dose corticosteroid use not reduced to physiologic levels.
  • Prior head or neck chemotherapy or radiotherapy except TMZ during radiation.
  • Severe active co-morbidities including unstable heart disease, infections, respiratory illness, hepatic insufficiency, autoimmune diseases requiring immunosuppression, or psychiatric impairments.
  • Pregnancy or breastfeeding.
  • History of brachial neuritis or Guillain-Barré syndrome.
  • Use of other investigational agents within 30 days.
  • Unwillingness or inability to receive treatment or follow-up.
  • Pediatric exclusions include diffuse intrinsic pontine glioma, bulky or extensive tumor involvement, extracranial disease, uncontrolled seizures, myocarditis, recent live vaccine, severe organ dysfunction, and unstable medical conditions.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 screening and enrollment visit

Surgery

Duration - Single event

Participants undergo surgery for tumor removal and collection of tumor material needed for RNA vaccine preparation.

1 visit (in-person)

Radiation

Duration - Duration as per standard of care

Participants receive standard radiation treatment as part of their care for glioblastoma or high-grade glioma.

Visit schedule depends on standard radiation therapy protocol

Immunotherapy

Duration - Up to 14 months

Participants receive RNA-lipid particle vaccines starting within 4 weeks after radiation. Initial vaccination includes three doses every 2 weeks followed by 12 monthly booster doses, totaling 15 vaccines over approximately 14 months.

Weekly visits during the initial 6 weeks for the first three vaccines, then monthly visits for up to 12 months

Follow-up

Duration - Up to 3 years post-immunotherapy

Participants are monitored after immunotherapy with MRI and clinical evaluations to assess disease progression and overall health.

MRI and clinical evaluations every 3 months for the first year, then every 6 to 12 months for up to 2 additional years

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

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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