Novel Molecular Targets and Innovative Therapeutic Perspective in Medulloblastoma

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.

NCT Number	NCT06959979
Lead Sponsor	Fondazione Policlinico Universitario Agostino Gemelli IRCCS Collaborators: University of Rome Tor Vergata, Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello, Università degli Studi di Enna Kore
Conditions	Medulloblastoma, Childhood
Enrollment	60 participants
Start Date	2024-11-25
Primary Completion	2026-08-31 (estimated)
Study Completion	2026-08-31 (estimated)
Updated on ClinicalTrials.gov	2025-05-07