NCT07492316

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

A Phase I/II Study of RNA-lipid Particle (RNA-LP) Vaccines for Newly Diagnosed Pediatric High-Grade Gliomas (pHGG) and Adult Glioblastoma (GBM), and Recurrent/Progressive Medulloblastoma (MB)

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: University of Florida · Academic / Other
• Sex: All
• Age: 4 Years – 39 Years
• Healthy volunteers: Not accepted

Summary

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)

Detailed description

This is a first in human Phase I study of RNA-LP vaccines for pediatric patients with recurrent/progressive Medulloblastoma (MB). The phase I portion of the study will involve a dose-escalation study to identify the maximally tolerated dose (MTD). This clinical trial will consist of three parts: Surgery, Salvage Therapy (Radiation and/or chemotherapy), and Immunotherapy. 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). Pediatric participants will have tumor material sent to the University of Florida (UF) from qualified PNOC sites. Following surgical resection with confirmatory pathologic diagnosis, patients will be enrolled in the trial after informed consent has been obtained. The RNA-LP vaccination will begin within 4 weeks following salvage therapy and after review of post-radiation MRI (for baseline). After radiation patients will receive three RNA-LP vaccines every 2 weeks before beginning 12 cycles of adjuvant monthly RNA- LP vaccines for a total of 15 vaccines. Participants may receive RNA-LP vaccines for up to 14 months. Participants will be followed until death due to any cause. MRI and clinical evaluation for assessment of disease progression will be conducted every 3 months for the first-year post-immunotherapy.

Conditions

• Recurrent Medulloblastoma

Interventions

Type	Name	Description
BIOLOGICAL	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)	RNA-LP vaccines will be administered intravenous. Three RNA-LP vaccines will be administered every 2 weeks followed by 12 cycles of adjuvant monthly RNA-LP vaccines for a total of 15 vaccines.

Timeline

• Start date: 2026-05-15
• Primary completion: 2030-09-01
• Completion: 2031-03-31
• First posted: 2026-03-25
• Last updated: 2026-03-25

Regulatory

• FDA-regulated drug study