Actively Recruiting

Phase 1
Age: 4Years - 39Years
All Genders
NCT07492316

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

Led by University of Florida · Updated on 2026-04-28

24

Participants Needed

1

Research Sites

254 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 pediatric patients with recurrent/progressive Medulloblastoma (MB)

CONDITIONS

Official Title

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

Who Can Participate

Age: 4Years - 39Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age greater than 3 years and up to 39 years
  • Histologically confirmed or suspected recurrent/progressive Medulloblastoma in first or second relapse
  • Prior radiation therapy received as part of treatment
  • Sterile collection of tumor material suitable for RNA extraction and loading into lipid particles
  • Fully recovered from acute toxic effects of prior cancer therapies with required minimum waiting periods
  • No prior exposure to pp65-directed therapy or RNA-LP therapy
  • Preoperative brain and spine MRI within 28 days before enrollment and postoperative MRI within 7 days after surgery
  • Karnofsky score of 60 or higher for participants older than 16 years, Lansky score of 60 or higher for participants younger than 16 years
  • Adequate bone marrow function (ANC ≥ 1,000/μl, platelets ≥ 100/μl unsupported for at least 7 days, hemoglobin > 8 g/dL)
  • Adequate kidney function (creatinine clearance or GFR ≥ 70 mL/min/1.73 m2)
  • Adequate liver function (bilirubin ≤ 3 times upper limit, ALT and AST ≤ 5 times upper limit)
  • Stable or decreasing corticosteroid dose for more than 1 week prior to enrollment, with dose limits
  • Willingness to take antiepileptic medication like levetiracetam during vaccinations
  • Ability to understand and sign informed consent
  • Negative pregnancy test at enrollment for women of childbearing potential
  • Willingness to use contraception during the study and for 24 weeks after last dose
  • Stable neurological deficits for at least 1 week prior to enrollment
  • Enrollment in PNOC COMP if available at the institution
Not Eligible

You will not qualify if you...

  • Diagnosis of diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E positive tumors
  • Bulky disease with significant brain or spine mass effect, extensive multilobular involvement (>3 lobes), or extracranial disease except spinal metastases in certain cases
  • Known HIV, Hepatitis B, or Hepatitis C infection
  • Uncontrolled seizure disorder
  • History of myocarditis
  • Receipt of any live vaccine within 30 days prior to enrollment
  • Known active infection or immunosuppressive disease
  • Significant organ dysfunction including renal, cardiac, pulmonary, or hepatic impairment
  • Severe or unstable concurrent medical conditions
  • Pregnancy or breastfeeding
  • Use of other investigational agents or therapeutic protocols within 30 days prior to enrollment
  • Inability or unwillingness to receive treatment and complete follow-up evaluations

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 Shands Children's Hospital

Gainesville, Florida, United States, 32608

Actively Recruiting

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Research Team

E

Elias Sayour, MD, PhD

CONTACT

J

Jannerfer An

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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