Pediatric Brain TumorsNCT07417072
GenoDrugP 2025: Study on Three-dimensional Models Derived From Brain Tumors in Pediatric Patients
A study testing Genomic DNA analysis of biological samples in people with Pediatric Brain Tumors and Genomics, run by Meyer Children's Hospital IRCCS at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Pediatric Brain Tumors, Genomics, Epigenetic
- What is being tested?
- Genomic DNA analysis of biological samples
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteItaly
- Study dates
- 21 Oct 2025to Oct 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Meyer Children's Hospital IRCCS
Florence, Firenze
Sardi, 0555662631, iacopo.sardi@meyer.it
About this study
Official 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.
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
- Being tested
- Genomic DNA analysis of biological samples
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Genomic DNA analysis of biological samplesDiagnostic test
Analysis of genomic DNA from tumor biopsy and blood samples
| Study group | Role | What they receive |
|---|---|---|
| Pediatric patients with brain tumors | Experimental | Not described Receives: Diagnostic Test: Genomic DNA analysis of biological samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Single Nucleotide Variants (SNV)
Number of SNVs germline in DNA from tumor and blood samples
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: At enrollment and on the date of first documented progression assessed up to 12 months
Vitality of three-dimensional models derived from the tumour
Measured: 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
Measured: At enrollment and on the date of first documented progression assessed up to 12 months
Percentage of residual cell vitality after drug treatment
Measured: 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
Measured: At enrollment and on the date of first documented progression assessed up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2026
- Study start21 Oct 2025
- Primary completionJun 2026 (estimated)
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Brain TumorsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Meyer Children's Hospital IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Iacopo Sardi, Meyer Children's Hospital IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07417072
- Sponsor's protocol ID
- GenoDrugP 2025
- Status last verified
- Feb 2026
- First submitted
- 2 Feb 2026
- Last updated
- 18 Feb 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT07417072
- Acronym / sponsor ID
- GenoDrugP 2025
- Conditions
- Pediatric Brain Tumors, Genomics, Epigenetic
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Meyer Children's Hospital IRCCSUniversity, hospital or other
- Study start
- 21 Oct 2025 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Oct 2026 (estimated)
- First posted
- 18 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric patients with brain tumors | Experimental | Diagnostic Test: Genomic DNA analysis of biological samples |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Genomic DNA analysis of biological samplesAnalysis of genomic DNA from tumor biopsy and blood samples | Diagnostic test | Pediatric patients with brain tumors |
Primary outcome
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
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
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
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
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
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
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
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
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Genomic DNA analysis of biological samples in people with Pediatric Brain Tumors and Genomics, run by Meyer Children's Hospital IRCCS at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Iacopo Sardi (0555662631, iacopo.sardi@meyer.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Single Nucleotide Variants (SNV) (time frame: At enrollment and on the date of first documented progression assessed up to 12 months). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2026.