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)
University of Florida
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)
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 pa…
Eligibility
- Age range
- 4–39 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age \> 3 and \</= 39 years. * Histologically confirmed or suspected recurrent/progressive MB in first or second relapse. * Patients must have received radiation therapy as part of prior therapy. * Patient must have been enrolled on a screening consent and have had sterile collection of tumor material in a manner suitable for RNA extraction, amplification, and loading of lipid particles (LPs). * Prior Therapy: Patients must have fully recovered from all acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer…
Interventions
- BiologicalAutologous 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.
Location
- UF Health Shands Children's HospitalGainesville, Florida