Study on RNA Lipid Particles for Treating Recurrent Brain and Other Solid Tumors in Children

Program Overview

This study is testing a new treatment using RNA lipid particles (RNA-LPs) to help the immune system fight against recurring brain tumors and other solid tumors in children. Researchers have found that these particles can activate the body's immune cells and help them target cancer cells. The study will look at how safe this treatment is and how well it works in children with these types of cancers.

Description

The study involves two main groups of patients: those with recurring high-grade brain tumors (pHGG) and those with recurring or hard-to-remove osteosarcoma (OSA). For brain tumors, there are two treatment options: one where the RNA-LP vaccine is given before surgery, and another where it is given after surgery. For osteosarcoma, patients are divided into three groups based on their cancer's location and spread.

The study will start with a small group of patients to determine the safest dose of the RNA-LP vaccine. This phase will include 18 patients, and both brain tumor and osteosarcoma groups will be monitored together for safety.

Participants must meet certain health criteria, such as having a specific type of brain tumor or osteosarcoma, being between 3 and 25 years old for brain tumors, and between 3 and 39 years old for osteosarcoma. They must have already tried standard treatments like surgery and radiation, and their cancer must have returned or continued to grow.

Patients will need to provide a tumor sample for the study, and they must be in good overall health, with adequate blood counts and organ function. Women who can become pregnant and men with partners who can become pregnant must use effective birth control during the study and for 24 weeks after the last dose of the study drug.

All participants and their guardians must understand and agree to the study's requirements and sign a consent form.

Eligibility Criteria

Locations