GenoDrugP 2025: Study on Three-dimensional Models Derived From Brain Tumors in Pediatric Patients

NCT07417072 | Recruiting

• 1 other identifier: GenoDrugP 2025
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Central nervous system tumours are the most common solid tumours and the leading cause of cancer mortality in children, with high biological and prognostic heterogeneity. Despite advances in the 2021 WHO molecular classifications, treatment options remain limited and often ineffective in high-grade tumours. New third-generation sequencing technologies and three-dimensional models derived from patient tumours offer promising tools for more comprehensive genomic characterisation and preclinical evaluation of drug responses. However, the lack of integrated preclinical studies remains a limitation, necessitating coordinated projects to develop personalised therapeutic strategies. The study aims to investigate the genetic and biological characteristics of paediatric brain tumours. To this end, tumour tissue samples taken during planned surgery and peripheral blood samples will be analysed. Advanced genetic analyses will be performed on these materials to identify tumour alterations and the patient's genetic characteristics. In addition, experimental in vitro models derived from the tumour will be developed to evaluate the response to different chemotherapy drugs. The information obtained will be used to better understand the mechanisms of tumour growth and resistance and to promote the future development of more targeted and personalised therapies.

Conditions

Pediatric Brain Tumors; Genomics; Epigenetic

Outcome Measures

Primary Outcomes (9)

• Number of Single Nucleotide Variants (SNV)

  Number of SNVs germline in DNA from tumor and blood samples

  At enrollment and on the date of first documented progression assessed up to 12 months

• Number of copy number variations (CNVs)

  Number of copy number variations (CNVs) in DNA from tumor and blood samples

  At enrollment and on the date of first documented progression assessed up to 12 months

• Number of triplet expansions

  Number of triplet expansions in DNA from tumor and blood samples

  At enrollment and on the date of first documented progression assessed up to 12 months

• Number of structural variants (SVs)

  Number of structural variants (SVs) in DNA from tumor and blood sample

  At enrollment and on the date of first documented progression assessed up to 12 months

• Morphological description of three-dimensional models derived from the tumour

  At enrollment and on the date of first documented progression assessed up to 12 months

• Vitality of three-dimensional models derived from the tumour

  At enrollment and on the date of first documented progression assessed up to 12 months

• Proliferative activity of three-dimensional models derived from the tumour

  At enrollment and on the date of first documented progression assessed up to 12 months

• Percentage of residual cell vitality after drug treatment

  At enrollment and on the date of first documented progression assessed up to 12 months

• Dose-response curves for each drug tested

  Ex vivo chemosensitivity study on three-dimensional models derived from primary tumour cells

  At enrollment and on the date of first documented progression assessed up to 12 months

Study Arms (1)

Pediatric patients with brain tumors

EXPERIMENTAL

Diagnostic Test: Genomic DNA analysis of biological samples

Interventions

Genomic DNA analysis of biological samples DIAGNOSTIC_TEST

Analysis of genomic DNA from tumor biopsy and blood samples

Pediatric patients with brain tumors

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients aged 3-18 years with suspected brain tumours undergoing neurosurgery
• No previous bone marrow transplants or other haematological procedures that could potentially interfere with germline analysis.
• Patients who have not received any systemic anticancer treatment (including chemotherapy, radiotherapy or targeted therapies) prior to enrolment surgery.
• Signature of informed consent

You may not qualify if:

• Subsequent histological confirmation of non-neoplastic brain pathology (e.g. malformations, inflammatory lesions, demyelinating processes).
• Insufficient quantity or quality of tumour tissue or peripheral blood for the analyses required by the protocol.

Sponsors & Collaborators

• Meyer Children's Hospital IRCCS: lead

Study Sites (1)

Meyer Children's Hospital IRCCS

Florence, Firenze, Italy

RECRUITING

Central Study Contacts

Iacopo Sardi

CONTACT

0555662631; iacopo.sardi@meyer.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: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: February 2, 2026
• First Posted: February 18, 2026
• Study Start: October 21, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)