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Targeting MYC in High-Risk Medulloblastoma

NCT07694622 · Status: NOT_YET_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2026-07-10

No results posted yet for this study

Summary

Medulloblastoma is the most common malignant brain tumor in children. Group 3 medulloblastoma (G3 MB) represents the most aggressive molecular subtype and is associated with poor prognosis, particularly in cases characterized by high expression or amplification of the MYC oncogene. Current treatment strategies are not tailored to this subgroup and are associated with significant long-term toxicities, highlighting the need for more specific therapeutic approaches.

This study aims to characterize biological processes and molecular pathways driven by high MYC expression in high-risk G3 medulloblastoma in order to identify potential therapeutic vulnerabilities. The study will investigate MYC-associated regulation of gene expression and RNA splicing in tumor cells and will define molecular dependencies that may be targeted using candidate or repurposed anticancer agents.

To achieve this, publicly available genomic datasets will be analyzed, findings will be validated in patient tumor specimens, and patient-derived three-dimensional (3D) tumor models will be established from surgical samples. These models will be used for ex vivo assessment of selected therapeutic strategies in a system that preserves key features of the original tumor.

This translational approach integrates computational analyses, molecular validation, and functional testing in patient-derived models to improve understanding of MYC-associated tumor biology in Group 3 medulloblastoma.

Conditions

Interventions

PROCEDURE

Tumor tissue collection

Tumor tissue will be collected during standard-of-care surgical resection of medulloblastoma. No additional surgical procedures will be performed for research purposes. Collected tissue will be used for molecular analyses and the establishment of patient-derived three-dimensional (3D) cultures for ex vivo translational research, including characterization of MYC-associated molecular pathways and evaluation of tumor cell responses to selected compounds in preclinical assays.

Sponsors & Collaborators

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    lead OTHER

Principal Investigators

  • Gianpiero Tamburrini · Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
20 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-01
Primary Completion
2028-09-01
Completion
2029-09-01

Countries

  • Italy

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07694622 on ClinicalTrials.gov