Pediatric Prospective Personalized Immune and Target Identification Trial

PPROSPERITIT will identify specific molecular changes by using genomic sequencing technologies in refractory/recurrent or very high-risk pediatric CNS tumors. The study will employ an analytically validated comparison of a selection of targeted agents/immune therapies on the basis of commercially available comprehensive genomic profiling FoundationOneHeme panel (F1Heme, comprising DNA and RNA analysis) vs selection of agents based on more complex DNA/RNA/Protein based analyses. This will be coupled to a computer algorithm that uses preexisting definitions and prioritization of target-agent pairs to assign patients by actionable mutation results to a targeted treatment. The selection of targeted agents will be performed by a multidisciplinary molecular tumor board, but the recommended treatment will not be a part of the PPROSPERTIT study.

Inclusion criteria

• Patient Informed consent form must be appropriately obtained under the applicable local and regulatory requirements. Each patient must sign a consent form prior the enrollment to document their willingness to participate.
• The subject is male or female, aged 1 - 19 years
• The subject must have a histologically proven recurrent/ refractory or very high-risk CNS tumors
• Patients must be in good overall physical condition, which allows tumor biopsy
• Patients must have a life expectancy of at least 3 months.
• Patients must have a tumor amenable to image-guided or direct vision biopsy and be willing and able to undergo a tumor biopsy and/or blood taking for molecular profiling.
• Patients must be accessible for follow-up.

Exclusion criteria

• Patients with known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Pregnant and/or breastfeeding women, if applicable
• No intention to treat the patient.