Glioma, MalignantPBTS23NCT06499636
In Vitro Models From Pediatric Brain Tumors
An observational study of Glioma, Malignant and Ependymoma, run by Fondazione Policlinico Universitario Agostino Gemelli IRCCS at 1 site in Italy.
Trial summary
Tumors of the Central Nervous System (CNS) represent the leading cause of cancer-related deaths in children. Current treatment options are not curative for most malignant histologies, and intense preclinical and clinical research are necessary to develop more effective therapeutic interventions against these tumors… Read more about this study
- Who is it for?
- Ages 1 year to 35 years
- Conditions the study is for
- Glioma, Malignant, Ependymoma, Medulloblastoma
- What is being tested?
- other
- How many people
- 100 participants
- Where
- 1 siteItaly
- Study dates
- 1 Sep 2024to 30 Jun 2029
- Phase
- Observational
- 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 1 year to 35 years
- All sexes
- Child, Adult
Study population: Children and young adults
Who may be able to join
- Children and young adults who undergo standard surgical resection for suspected primary brain tumors or recurrent brain tumors
- signed written informed consent
Who cannot join
- no informed consent
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
Pediatric Oncology Unit- Fondazione Policlinico Gemelli IRCCS
Roma, Rome, 00168
Antonio Ruggiero, MD, 0039063015155, antonio.ruggiero@policlinicogemelli.it
About this study
Official title: In Vitro Models Derived From Pediatric Glial Tumors and Pediatric Embryonal Tumors for Drug Testing and Molecular Studies
Tumors of the Central Nervous System (CNS) represent the leading cause of cancer-related deaths in children. Current treatment options are not curative for most malignant histologies, and intense preclinical and clinical research are necessary to develop more effective therapeutic interventions against these tumors, most of which meet the FDA definition for orphan diseases. The majority of malignant CNS tumors in children and adolescents belong to two broad histologic tumor entities: those of glial origin, such as high-grade glioma (HGG)and ependymoma (EPN), and those of neuronal origin, also identified as embryonal tumors, that include medulloblastoma and AT/RT(1). Over the last few years, whole-genome sequencing, gene-expression profiling and genome-wide methylation studies have greatly deepened our understanding of the biology and genetics of these tumors, allowing for robust stratification in clinically relevant molecular subgroups. The advancement of single-cell omics over the last decade have highlighted the enormous heterogeneity of tumors, a complex mixture of co-existing cancer subclones and supportive normal cell populations.
However, current treatments have remained largely static, and 5-year survival rate for children with malignant CNS tumors only achieves a modest 57.5%.
Read more
More effective treatment strategies should include novel chemotherapeutic agents that take into account high intrinsic tumor heterogeneity as well as the complex regulations of transcriptional and translational mechanisms that control protein expression. Identification of novel drugs and treatment strategies is further limited by the paucity of appropriate preclinical models, which mirror the molecular characteristics of distinct tumor subgroups.
We propose to establish patient-derived in vitro models to predict chemotherapeutic drug sensitivity/resistance in malignant pediatric CNS tumors. Next, we propose to perform molecular analyses in tissues of pediatric CNS tumors to determine whether in vitro findings have clinical correlates.
- Conditions
- Glioma, Malignant, Ependymoma, Medulloblastoma
- Being tested
- other
- Medical subject headings
- Glioma, Ependymoma, Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
otherOther
other
What is being measured
The primary outcome is the main question the study is designed to answer.
1) Establishment of in vitro models derived from pediatric tumors of the CNS, which more faithfully mirror the molecular features and heterogeneity of pediatric EPNs.
Collection and storage of snap-frozen pediatric brain tumor samples (tumor specimens and blood samples).
Generation of patient-derived cell lines.
Measured: 48 months
Secondary outcomes (2)
2) Preclinical drug testing, to explore the chemotherapeutic potential of selected standard and molecularly-targeted agents.
The models that will be prospectively established in the objective number 1 and the already commercially available models will be used for preclinical in vitro studies with agents belonging to imipridones and DHODH inhibitors
Measured: 24 months
3) Molecular studies on pediatric brain tumor tissues, to determine whether the cellular, molecular and biochemical findings in in vitro models have clinical correlates.
To determine whether the findings in preclinical models have clinical correlates, tumor samples that will prospectively be collected and stored in the Biobank of Fondazione Policlinico Universitario Gemelli will be analyzed for the expression of selected biomarkers. RNA extraction, reverse transcription and real-time PCR will be performed
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2024
- Study start1 Sep 2024 (estimated)
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Jun 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glioma, MalignantStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Antonio Ruggiero, prof, Principal investigator, Fondazione Policlinico Gemelli IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06499636
- Sponsor's protocol ID
- 6351
- Acronym
- PBTS23
- Status last verified
- Jun 2024
- First submitted
- 8 Jul 2024
- Last updated
- 12 Jul 2024
- 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 12 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- In Vitro Models Derived From Pediatric Glial Tumors and Pediatric Embryonal Tumors for Drug Testing and Molecular Studies
- ClinicalTrials.gov ID
- NCT06499636
- Acronym / sponsor ID
- PBTS23 / 6351
- Conditions
- Glioma, Malignant, Ependymoma, Medulloblastoma
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCSUniversity, hospital or other
- Study start
- 1 Sep 2024 (estimated)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Jun 2029 (estimated)
- First posted
- 12 Jul 2024 (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.
Tumors of the Central Nervous System (CNS) represent the leading cause of cancer-related deaths in children. Current treatment options are not curative for most malignant histologies, and intense preclinical and clinical research are necessary to develop more effective therapeutic interventions against these tumors, most of which meet the FDA definition for orphan diseases. The majority of malignant CNS tumors in children and adolescents belong to two broad histologic tumor entities: those of glial origin, such as high-grade glioma (HGG)and ependymoma (EPN), and those of neuronal origin, also identified as embryonal tumors, that include medulloblastoma and AT/RT(1). Over the last few years, whole-genome sequencing, gene-expression profiling and genome-wide methylation studies have greatly deepened our understanding of the biology and genetics of these tumors, allowing for robust stratification in clinically relevant molecular subgroups. The advancement of single-cell omics over the last decade have highlighted the enormous heterogeneity of tumors, a complex mixture of co-existing cancer subclones and supportive normal cell populations.
However, current treatments have remained largely static, and 5-year survival rate for children with malignant CNS tumors only achieves a modest 57.5%.
More effective treatment strategies should include novel chemotherapeutic agents that take into account high intrinsic tumor heterogeneity as well as the complex regulations of transcriptional and translational mechanisms that control protein expression. Identification of novel drugs and treatment strategies is further limited by the paucity of appropriate preclinical models, which mirror the molecular characteristics of distinct tumor subgroups.
We propose to establish patient-derived in vitro models to predict chemotherapeutic drug sensitivity/resistance in malignant pediatric CNS tumors. Next, we propose to perform molecular analyses in tissues of pediatric CNS tumors to determine whether in vitro findings have clinical correlates.
Study design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Ages
- 1 year to 35 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Children and young adults
Key inclusion criteria (2 listed)
- Children and young adults who undergo standard surgical resection for suspected primary brain tumors or recurrent brain tumors
- signed written informed consent
Key exclusion criteria (1 listed)
- no informed consent
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 |
|---|---|---|
| otherother | Other | – |
Primary outcome
1) Establishment of in vitro models derived from pediatric tumors of the CNS, which more faithfully mirror the molecular features and heterogeneity of pediatric EPNs.
Time frame: 48 months
Collection and storage of snap-frozen pediatric brain tumor samples (tumor specimens and blood samples). Generation of patient-derived cell lines.
2 secondary outcomes
2) Preclinical drug testing, to explore the chemotherapeutic potential of selected standard and molecularly-targeted agents.
Time frame: 24 months
3) Molecular studies on pediatric brain tumor tissues, to determine whether the cellular, molecular and biochemical findings in in vitro models have clinical correlates.
Time frame: 36 months
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 12 Jul 2024.
- 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.
Antonio Ruggiero, prof principal investigator
Fondazione Policlinico Gemelli IRCCS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glioma, Malignant and Ependymoma, run by Fondazione Policlinico Universitario Agostino Gemelli 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 2 inclusion and 1 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: opening soon, as of the sponsor's last update on 12 Jul 2024. 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 Antonio Ruggiero, prof (00390630155155, antonio.ruggiero@unicatt.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1) Establishment of in vitro models derived from pediatric tumors of the CNS, which more faithfully mirror the molecular features and heterogeneity of pediatric EPNs. (time frame: 48 months). 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 12 Jul 2024.