An observational study in Glioma, Malignant, Ependymoma and Medulloblastoma, sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS. Not yet recruiting at 1 site in Italy. Open to participants aged 1 Year to 35 Years. Per ClinicalTrials.gov, last updated 2024-07-12.
Sponsored by Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Observational
Tumors of the Central Nervous System (CNS) represent the leading cause of cancer-related deaths in children. Current treatment options are not curative for most malignant histologies, and intense preclinical and clinical research are necessary to develop more effective therapeutic interventions against these tumors, most of which meet the FDA definition for orphan diseases. The majority of malignant CNS tumors in children and adolescents belong to two broad histologic tumor entities: those of glial origin, such as high-grade glioma (HGG)and ependymoma (EPN), and those of neuronal origin, also identified as embryonal tumors, that include medulloblastoma and AT/RT(1). Over the last few years, whole-genome sequencing, gene-expression profiling and genome-wide methylation studies have greatly deepened our understanding of the biology and genetics of these tumors, allowing for robust stratification in clinically relevant molecular subgroups. The advancement of single-cell omics over the last decade have highlighted the enormous heterogeneity of tumors, a complex mixture of co-existing cancer subclones and supportive normal cell populations.
However, current treatments have remained largely static, and 5-year survival rate for children with malignant CNS tumors only achieves a modest 57.5%.
More effective treatment strategies should include novel chemotherapeutic agents that take into account high intrinsic tumor heterogeneity as well as the complex regulations of transcriptional and translational mechanisms that control protein expression. Identification of novel drugs and treatment strategies is further limited by the paucity of appropriate preclinical models, which mirror the molecular characteristics of distinct tumor subgroups.
We propose to establish patient-derived in vitro models to predict chemotherapeutic drug sensitivity/resistance in malignant pediatric CNS tumors. Next, we propose to perform molecular analyses in tissues of pediatric CNS tumors to determine whether in vitro findings have clinical correlates.
1,452 studies on the registry are indexed under Glioma; 349 are open to participants now.
This study's planned enrollment of 100 is above the median of 90 across 273 observational studies indexed under Glioma.
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Children and young adults
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
other
1) Establishment of in vitro models derived from pediatric tumors of the CNS, which more faithfully mirror the molecular features and heterogeneity of pediatric EPNs.
Collection and storage of snap-frozen pediatric brain tumor samples (tumor specimens and blood samples). Generation of patient-derived cell lines.
Time frame: 48 months
2) Preclinical drug testing, to explore the chemotherapeutic potential of selected standard and molecularly-targeted agents.
The models that will be prospectively established in the objective number 1 and the already commercially available models will be used for preclinical in vitro studies with agents belonging to imipridones and DHODH inhibitors
Time frame: 24 months
3) Molecular studies on pediatric brain tumor tissues, to determine whether the cellular, molecular and biochemical findings in in vitro models have clinical correlates.
To determine whether the findings in preclinical models have clinical correlates, tumor samples that will prospectively be collected and stored in the Biobank of Fondazione Policlinico Universitario Gemelli will be analyzed for the expression of selected biomarkers. RNA extraction, reverse transcription and real-time PCR will be performed
Time frame: 36 months
Plan to share: No
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This study is not yet recruiting, as verified in Jun 2024. You cannot join it, but the record below documents what was studied.
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