SIOPEN BIOPORTAL, An International Registry Linked to a Virtual Biobank for Patients With Peripheral Neuroblastic Tumours

Study Overview
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Brief Summary
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The SIOPEN BIOPORTAL is a prospective non-therapeutic multi-centre international study aimed at developing an international Registry linked to a Virtual Biobank for all the patients with peripheral neuroblastic tumor within countries of the SIOPEN network. The overall aim of this study is to provide a GDPR-compliant framework to collect basic clinical annotations, biological and genetic features and information about the location on biospecimens for all the patients with a peripheral neuroblastic tumor including neuroblastoma, ganglioneuroblastoma and ganglioneuroma in the SIOPEN network. This study will support data and sample management and intensify cross-borders data and sample sharing fostering translational and clinical research. The post-hoc hypothesis formulated based on the data generated in this study will be used as statistical basis for future precision medicine programs based on improved biological characterization, patient stratification and therapeutic management.

Official Title
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SIOPEN BIOPORTAL, An International Registry Linked to a Virtual Biobank for Patients With Peripheral Neuroblastic Tumours

Conditions
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Neuroblastoma, Ganglioneuroblastoma, Ganglioneuroma

Intervention / Treatment
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* Other: No Intervention NA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: I-1. Patients with PNT: ganglioneuroma or ganglioneuroblastoma or neuroblastoma, I-2. At the time of initial diagnosis of neuroblastic tumour, or at relapse/progression if not yet registered I-3. Written patient informed consent, or parents or legal representative written informed consent and assent of the child, adolescent or young adult Non-Inclusion Criteria: R-1. (Adult) Patients with olfactory neuroblastoma R-2. Central nervous system (CNS) neuroblastoma (as per the WHO classification)
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: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Peripheral neuroblastic tumors (ganglioneuroma, ganglioneuroblastoma and neuroblastoma)<br> | Other: No Intervention NA<br>* Additional blood sampling (not mandatory)<br>* Other names: Bio-sampling;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of patients having their clinical data registered | Registry Outcome: Rate of patients having their clinical data registered throughout their treatment and follow-up from the total of patients registered | 6 months | 
| Rate of patients for whom information on biological samples/material is collected | Virtual Biobank Outcome: Rate of patients for whom information on biological samples/material collected and stored in SIOPEN is available from the total of patients registered | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival (PFS) | Investigation of the impact of the variables currently used for assignment to risk groups and treatment stratification (MYCN Status, ALK Status, stage, age, primary tumor site) on progression-free survival (PFS) using the Kaplan-Meier method | 12 months | 
| Overall-survival (OS) | Investigation of the impact of the variables currently used for assignment to risk groups and treatment stratification (MYCN Status, ALK Status, stage, age, primary tumor site) on overall-survival (OS) using the Kaplan-Meier method | 12 months |