Ignite Creation Date:
2024-07-17 @ 11:41 AM
Last Modification Date:
2024-10-26 @ 3:34 PM
Study NCT ID:
NCT06499636
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-07-12
First Post:
2024-07-08
Brief Title:
In Vitro Models From Pediatric Brain Tumors
Sponsor:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Organization:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Study Overview
Official Title:
In Vitro Models Derived From Pediatric Glial Tumors and Pediatric Embryonal Tumors for Drug Testing and Molecular Studies
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PBTS23
Brief Summary:
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 HGGand ependymoma EPN and those of neuronal origin also identified as embryonal tumors that include medulloblastoma and ATRT1 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 575
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 sensitivityresistance 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
However current treatments have remained largely static and 5-year survival rate for children with malignant CNS tumors only achieves a modest 575
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 sensitivityresistance 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None