Pediatric Prospective Personalized Immune and Target Identification Trial

Study Overview
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Brief Summary
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PPROSPERITIT is a prospective clinical study assessing the use of comprehensive molecular profiling to define the best matching targeted and immune treatment for relapsed, refractory or very high risk pediatric CNS tumors.

Detailed Description
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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.

Official Title
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Prospective, Interventional Diagnostic, Multicenter, Non-treatment Clinical Study Identifying Specific Molecular Changes by Using Genomic Sequencing Technologies in Refractory/Recurrent or Very High-risk Pediatric CNS Tumors.

Conditions
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Central Nervous System Neoplasms, Pediatric Cancer, GEN1 Gene Mutation, Tumor, Brain

Intervention / Treatment
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* Device: FoundationOneHeme


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 19 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Diagnostic group<br>Tumor and blood samples will be collected from each patient and broad molecular profiling will be performed. The results of the evaluation of the tumor specimens will determine if the patient's tumor has an actionable mutation for which treatment is available. | Device: FoundationOneHeme<br>* Tumor tissue obtained during standard surgery will be subsequently examined histopathologically and the content of cancer cells will be determined. Broad molecular profiling of the tumor; with potential results finding such molecular changes, for which the specific targeted anti-tumor treatment will be performed.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The proportion of patients in which F1Heme molecular testing identified at least 1 clinically relevant alteration at the time of MTB decision. | Evaluation of the feasibility of FoundationOneHeme | Diagnostic assessment is done within 28 days from enrolment patient in the study. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatric oncology, CNS tumors, Personalized medicine