Targeting MYC in High-Risk Medulloblastoma

NCT07694622 | Not Yet Recruiting

• 1 other identifier: 27529
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief 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

Medulloblastoma

Keywords

Medulloblastoma; Group 3 Medulloblastoma; MYC; RNA Splicing; Patient-Derived 3D Cultures; Gene Expression; MYC Amplification; Organoids; Transcriptomics

Outcome Measures

Primary Outcomes (1)

• Successful establishment of patient-derived organoids (PDOs) from medulloblastoma surgical specimens

  Rate of successful generation of patient-derived organoids (PDOs) from freshly resected high-risk Group 3 medulloblastoma surgical specimens. Success will be defined by the ability of tumor samples to generate stable and expandable three-dimensional (3D) cultures in vitro. Established models will be evaluated for histological and molecular concordance with the corresponding parental tumors using standard pathological and genetic analyses.

  Up to 24 months

Study Arms (1)

Medulloblastoma tissue collection and ex vivo translational research

EXPERIMENTAL

Participants with medulloblastoma undergoing standard-of-care surgical resection will be enrolled after informed consent. Tumor tissue collected during surgery will be used to generate patient-derived organoids (PDOs) for ex vivo translational research. Analyses will include molecular characterization, transcriptomic profiling, and evaluation of tumor cell responses to selected anticancer compounds in preclinical assays. No investigational drugs or medical devices are administered to participants, and no additional clinical procedures beyond routine care are performed.

Procedure: Tumor tissue collection

Interventions

Tumor tissue collection PROCEDURE

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.

Medulloblastoma tissue collection and ex vivo translational research

Eligibility Criteria

• Age: Up to 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with histologically confirmed Group 3 medulloblastoma (G3 MB)
• Age between 0 and 20 years
• Undergoing surgical treatment at the UOC Child Neurosurgery Unit, Fondazione Policlinico Universitario "A. Gemelli", IRCCS, Rome
• Availability of tumor tissue obtained during standard-of-care surgical resection for molecular analyses and/or establishment of patient-derived organoids
• Written informed consent provided by the patient and/or parent/legal guardian (prospective cohort)

You may not qualify if:

• None

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Study Sites (1)

Fondazione Policlinico Universitario "A. Gemelli", IRCCS - UOC Neurochirurgia Infantile

Rome, Lazio, 00168, Italy

Location

MeSH Terms

Conditions

Medulloblastoma

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neuroectodermal Tumors, Primitive; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Gianpiero Tamburrini

  Fondazione Policlinico Universitario Agostino Gemelli IRCCS

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gianpiero Tamburrini

CONTACT

+393349468776; gianpiero.tamburrini@policlinicogemelli.it

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a single-group translational study involving analysis of tumor specimens collected during standard-of-care surgical procedures. The study includes retrospective and prospective cohorts. No investigational drugs or medical devices are administered. Biological samples are used for molecular analyses and the establishment of patient-derived three-dimensional (3D) models for ex vivo functional studies.

Study Record Dates

• First Submitted: July 3, 2026
• First Posted: July 10, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2029
• Last Updated: July 10, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)