Novel Molecular Targets and Innovative Therapeutic Perspective in Medulloblastoma

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Early Phase 1
SUMMARY

Medulloblastoma (MB), a rare yet critical pediatric brain tumor, is divided into 4 molecular subgroups (WNT, SHH, Group 3, Group 4), each with distinct genetic profiles. Despite diagnostic and therapeutic advances, neurotoxicity from standard treatments (resection, radiotherapy, chemotherapy) and the need for long-term care remain challenges. CDK4/6 inhibitors (palbociclib, ribociclib, abemaciclib), approved for breast cancer, show potential in other tumors, but their efficacy in MB is unclear. Treatment resistance is a concern. This project aims to identify genetic markers of sensitivity to CDK4/6 inhibitors in MB, to improve therapies and overcome resistance.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: f
View:

• All patients affected by Medulloblastoma operated on at the Pediatric Neurosurgery Unit during their pediatric age will be elected to take part to the study

Locations
Other Locations
Italy
Fondazione Policlinico Gemelli Irccs
RECRUITING
Roma
Contact Information
Primary
GIANPIERO TAMBURRINI, MD
gianpiero.tamburrini@unicatt.it
3349468776
Backup
MARIA CONCETTA GELOSO, PHD
mariaconcetta.geloso@unicatt.it
Time Frame
Start Date: 2024-11-25
Estimated Completion Date: 2026-08-31
Participants
Target number of participants: 60
Treatments
Experimental: Prospective
To establish ex-vivo translational models of MB, we will develop patient-derived organoids (PDOs) from primary surgical biopsies of patients
Experimental: Retrospective
To establish ex-vivo translational models of MB, we will develop patient-derived organoids (PDOs) from primary surgical biopsies of patients
Sponsors
Collaborators: Università degli Studi di Enna Kore, Università degli studi di Roma Tor Vergata, Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
Leads: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

This content was sourced from clinicaltrials.gov

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