In Vitro Models From Pediatric Brain Tumors

NCT ID: NCT06499636

Last Updated: 2024-07-12

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-09-01
• Study Completion Date: 2029-06-30

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 (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.

Conditions

Glioma, Malignant; Ependymoma; Medulloblastoma

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Interventions

other

other

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Children and young adults who undergo standard surgical resection for suspected primary brain tumors or recurrent brain tumors
• signed written informed consent

Exclusion Criteria

• no informed consent

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 35 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Antonio Ruggiero, prof

Role: PRINCIPAL_INVESTIGATOR

Fondazione Policlinico Gemelli IRCCS

Locations

Pediatric Oncology Unit- Fondazione Policlinico Gemelli IRCCS

Roma, Rome, Italy

Countries

Italy

Central Contacts

Antonio Ruggiero, prof

Role: CONTACT

antonio.ruggiero@unicatt.it

00390630155155

Tiziana Servidei, PhD

Role: CONTACT

tiziana.servidei@guest.policlinicogemelli.it

00390630155155

Facility Contacts

Antonio Ruggiero, MD

Role: primary

antonio.ruggiero@policlinicogemelli.it

0039063015155

Other Identifiers

6351

Identifier Type: -

Identifier Source: org_study_id