Novel Molecular Targets and Innovative Therapeutic Perspective in Medulloblastoma

Sponsor
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Study ID
NCT06959979
Phase
EARLY_PHASE1
Status
Recruiting
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Conditions

  • Medulloblastoma, Childhood

Eligibility Criteria

Sex
ALL
Age
0 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Nov 25, 2024
Status verified
Jul 2024
Primary completion
Aug 31, 2026
Completion
Aug 31, 2026

Study Design

Enrollment
60 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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

Primary Outcome Measure

Clarify the molecular mechanisms underlying the acquisition of resistance to CDK4/6i in MB. [ Time Frame: 2 yrs ]

Central Contacts

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