GenoDrugP 2025: Study on Three-dimensional Models Derived From Brain Tumors in Pediatric Patients
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-18
SponsorMeyer Children's Hospital IRCCS
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Genomic DNA analysis of biological samples
Diagnostic testAnalysis of genomic DNA from tumor biopsy and blood samples
Treatment groups
50 Participants
are divided into 1 treatment groupGroup A: Pediatric patients with brain tumorsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Number of Single Nucleotide Variants (SNV)
Number of SNVs germline in DNA from tumor and blood samples
Time frame
At enrollment and on the date of first documented progression assessed up to 12 months2
Number of copy number variations (CNVs)
Number of copy number variations (CNVs) in DNA from tumor and blood samples
Time frame
At enrollment and on the date of first documented progression assessed up to 12 months3
Number of triplet expansions
Number of triplet expansions in DNA from tumor and blood samples
Time frame
At enrollment and on the date of first documented progression assessed up to 12 months4
Number of structural variants (SVs)
Number of structural variants (SVs) in DNA from tumor and blood sample
Time frame
At enrollment and on the date of first documented progression assessed up to 12 monthsSponsors and contacts
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Source ClinicalTrials.gov