Actively Recruiting
A Phase I/II Study of RNA-lipid Particle Vaccines for Pediatric High-Grade Gliomas and Recurrent/Progressive Medulloblastoma
Led by University of Florida · Updated on 2026-04-28
24
Participants Needed
1
Research Sites
30 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
Researchers are studying RNA-lipid Particle (RNA-LP) vaccines in pediatric patients with recurrent or progressive Medulloblastoma (MB). This first-in-human Phase I trial aims to show that these vaccines can be safely manufactured and to find the maximum tolerated dose (MTD). The study focuses on children and young adults who have had prior radiation therapy and have recurrent MB. The study includes three parts: surgery to collect tumor tissue, salvage therapy with radiation and/or chemotherapy, and immunotherapy using the RNA-LP vaccines. After surgical tumor removal and confirmation of diagnosis, patients start vaccination within 4 weeks following salvage therapy and a baseline MRI. Vaccines are given intravenously with three doses every two weeks followed by 12 monthly doses, totaling 15 vaccines over about 14 months. Participants will be monitored regularly with MRI and clinical evaluations every three months for one year after immunotherapy to assess disease progression. The study tracks vaccine manufacturing feasibility, safety within the first three vaccine doses, and determines the MTD over up to five years. Participants will be followed until death or study end, with ongoing clinical and imaging assessments throughout the trial.
CONDITIONS
Brief Title
RNA-lipid Particle (RNA-LP) Vaccines for Recurrent/Progressive Medulloblastoma (MB)
Who Can Participate
Eligibility Criteria
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 completed as part of treatment
- Enrollment on screening consent with sterile tumor material collected for RNA processing
- Fully recovered from acute toxic effects of prior anti-cancer therapy, meeting required waiting periods
- Diagnostic contrast-enhanced MRI of brain and spine before and after surgery within specified timeframes
- Karnofsky or Lansky Performance Score of 60 or higher assessed within 2 weeks prior to enrollment
- Adequate bone marrow, renal, and liver function as defined by specified laboratory values
- Stable or decreasing steroid dose for more than 1 week before enrollment, with dose limits
- Willingness to take antiepileptic medication during vaccinations
- Ability to understand and sign informed consent
- Negative pregnancy test for women of childbearing potential at enrollment
- Willingness to use contraceptive methods for women and men of childbearing/fathering potential
- Stable post-surgical neurological deficits for at least 1 week
- Enrollment on PNOC COMP if open at the institution
You will not qualify if you...
- Diagnosis of diffuse intrinsic pontine glioma, brainstem diffuse midline glioma, or BRAFV600E positive tumor
- Bulky disease causing significant brain or spine mass effect or extensive multilobular involvement
- Tumor with extracranial disease except spinal metastases in specific strata
- Known positive status for HIV, Hepatitis B, or Hepatitis C
- Uncontrolled seizure disorder
- History of myocarditis
- Receipt of live vaccine within 30 days before enrollment
- Active infection or immunosuppressive disease
- Significant renal, cardiac, pulmonary, hepatic, or other organ dysfunction
- Severe or unstable concurrent medical conditions
- Pregnant or breast-feeding women
- Use of other investigational agents or therapeutic protocols within 30 days prior
- Unwillingness or inability to receive treatment and attend follow-up visits
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 week
Participants undergo surgical resection to collect tumor material for vaccine preparation.
1 visit (in-person)
Duration - Variable, up to 4 weeks
Participants receive radiation and/or chemotherapy as salvage therapy before immunotherapy.
Visit schedule depends on therapy received
Duration - Up to 14 months
Participants receive RNA-LP vaccines starting within 4 weeks after salvage therapy, with three vaccines given every 2 weeks followed by 12 monthly vaccines for up to 14 months.
15 vaccination visits over 14 months
Duration - 12 months
Participants are monitored with MRI and clinical evaluations every 3 months for the first year after immunotherapy to assess disease progression.
Quarterly visits for 12 months
Trial Site Locations
Total: 1 location
1
UF Health Shands Children's Hospital
Gainesville, Florida, United States, 32608
Actively Recruiting
Research Team
E
Elias Sayour, MD, PhD
J
Jannerfer An
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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