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NCT Number: NCT07417072

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

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.

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Key information

Conditions

Pediatric Brain Tumors Epigenetic Genomics

Age range

3 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Meyer Children's Hospital IRCCS

Florence, Firenze, Italy

Location status: Recruiting

Location contact

Sardi

CONTACT

[email protected]

0555662631

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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.
  • Presence of serious clinical conditions, systemic infections or haemodynamic instability that contraindicate the collection of biological samples or inclusion in the study.

Treatment and study plan

Genomic DNA analysis of biological samples

Diagnostic Test

Analysis of genomic DNA from tumor biopsy and blood samples

Primary outcomes

  1. Number of Single Nucleotide Variants (SNV)

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

    Number of SNVs germline in DNA from tumor and blood samples

  2. Number of copy number variations (CNVs)

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

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

  3. Number of triplet expansions

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

    Number of triplet expansions in DNA from tumor and blood samples

  4. Number of structural variants (SVs)

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

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

  5. Morphological description of three-dimensional models derived from the tumour

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

  6. Vitality of three-dimensional models derived from the tumour

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

  7. Proliferative activity of three-dimensional models derived from the tumour

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

  8. Percentage of residual cell vitality after drug treatment

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

  9. Dose-response curves for each drug tested

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

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

Study contacts

Contact information is provided by the study sponsor or research team.

Iacopo Sardi

CONTACT

[email protected]

0555662631

Sponsors and collaborators

Lead sponsor

Meyer Children's Hospital IRCCS

Other

Registry information

Official study title

Preliminary Study With Biological Samples, Single-center, Non-profit, to Identify Biological Mechanisms and Resistance to Therapies in Three-dimensional Models Derived From Brain Tumors in Pediatric Patients.

Important dates

Study start
2025
Primary completion
2026
Study completion
2026
First posted
Feb 18, 2026
Registry last updated
Feb 18, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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