MedulloblastomaISOPNCT06814353
Intraoperatively Observed Site of Origin and Growth Pattern of Medulloblastoma
An observational study of Medulloblastoma, run by Christian Dorfer at 4 sites in 3 countries.
Trial summary
The goal of this observational study is to provide accurate and systematic data on the site of origin and growth pattern of medulloblastoma from a neurosurgical perspective. By integrating intraoperative, radiological and genetic classification data, this study will contribute to our current understanding of the… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Medulloblastoma
- What is being tested?
- Intraoperative assessment of the site of origin and growth pattern
- Type of treatment
- Surgery or procedure
- How many people
- 100 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 1 Mar 2024to Mar 2026
- How long will I take part?
- 1 Year
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Both adult and pediatric patients who undergo surgical resection of a histologically confirmed medulloblastoma at a center with expertise in pediatric neurosurgery
Who may be able to join
- Patients who are referred for surgical resection of a medulloblastoma at a hospital with expertise in pediatric neurooncology
- Both pediatric patients (aged < 18 years at the time of diagnosis) and adult patients (aged > 18 years at the time of diagnosis)
Who cannot join
- Patients who are preoperatively admitted with severe tumor hemorrhage accompanied by clinical deterioration are excluded because these patients frequently do not receive magnetic resonance imaging as would be necessary for the present study, and the intraoperative assessment of the STO is limited in accuracy due to decreased visibility caused by the intra- and extratumoral hemorrhage.
- If an intraoperative complication occurs (e.g. bleeding) which impairs visibility of the neuroanatomic structures and does not allow an accurate assessment of the STO, the patient is excluded.
- If the histopathological and molecular analysis does not confirm the diagnosis of a MB, the patient has to be excluded as well.
Trial locations and contacts
4 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 3 countries
Germany2 sites
Department of Neurosurgery, Charité Berlin
Berlin
Section of Pediatric Neurosurgery, University Hospital of Tuebingen
Tübingen
Austria1 site
Department of Neurosurgery, Medical University of Vienna
Vienna, State of Vienna, 1090
Netherlands1 site
Department of Neurosurgery, Princess Maxima Centre for Pediatric Oncology
Utrecht
About this study
Official title: An International Multicenter Prospective Cohort Study Investigating the Intraoperatively Observed Site of Origin and Growth Pattern of the Molecular Groups of Medulloblastoma
The goal of this observational study is to provide accurate and systematic data on the site of origin and growth pattern of medulloblastoma from a neurosurgical perspective. By integrating intraoperative, radiological and genetic classification data, this study will contribute to our current understanding of the development, site of origin and growth pattern of medulloblastoma and advance the predictive accuracy of radiogenomics models. Patients with histologically confirmed medulloblastoma who undergo surgical resection at a high-volume center with expertise in pediatric neurosurgery will be included.
The main questions it aims to answer are:
Read more
- Is there a significant difference between the intraoperatively observed site of origin and the preoperatively or postoperatively radiologically assessed site of origin of medulloblastoma?
- How does the intraoperatively observed site of origin align with the site of origin associated with the molecular group based on the developmental cell lineage concept of medulloblastoma?
- Does incorporating the intraoperatively observed site of origin as a feature improve the predictive accuracy of radiomic models for molecular group classification?
Participants will:
- Undergo intraoperative assessment of site of origin and growth pattern by an experienced pediatric neurosurgeon.
- Have their site of origin and growth pattern evaluated on pre- and postoperative magnetic resonance imaging by an neuroradiologist with expertise in pediatric brain tumors.
- Conditions
- Medulloblastoma
- Being tested
- Intraoperative assessment of the site of origin and growth pattern
- Keywords
- Medulloblastoma
- Medical subject headings
- Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intraoperative assessment of the site of origin and growth patternProcedure or surgery
Also called Assessment of the site and origin and growth pattern on preoperative MRI, Evaluation of anatomic features, Imaging feature extraction and characterization, Histopathological and molecular analysis
The assessment of the epicenter and extension and therefore the assumed origin of tumor growth is conducted by an experienced pediatric neurosurgeon blinded to group allocation based on the intraoperative impression. The anatomical features and site of origin are systematically documented on the surgical form in all cases of this study.
| Study group | Role | What they receive |
|---|---|---|
| WNT-MB | Patients with a medulloblastoma of the subgroup MB, WNT(Wingless)-activated according to the WHO classification 2021. Receives: Procedure: Intraoperative assessment of the site of origin and growth pattern | |
| SHH-MB | Patients with a medulloblastoma of the subgroup MB, SHH (Sonic Hedgehog)-activated according to the World Health Organization (WHO) classification 2021. This subgroup can be further differentiated into SHH-activated (SHH-MB) TP53-wildtype and MB, SHH-activated TP53-mutant. Receives: Procedure: Intraoperative assessment of the site of origin and growth pattern | |
| Group 3 and Group 4 | Patients with a medulloblastoma of the subgroup MB, non-WNT/non-SHH activated MB (Group 3 and 4) according to the WHO classification 2021. Receives: Procedure: Intraoperative assessment of the site of origin and growth pattern |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of agreement between the intraoperatively observed site of origin and preoperatively radiologically assessed site of origin of medulloblastoma
The intraoperatively observed site of origin is categorized into brainstem, cerebellar hemispheres and cerebellar vermis.The intraoperatively observed site of origin is assessed by an experienced pediatric neurosurgeon blinded to subgroup allocation and systemically documented in surgical forms.
The location and site of origin is assessed by one expert neuroradiologist with expertise in pediatric brain tumor imaging who is blinded to the group allocation.
Preoperative magnetic resonance imaging (MRI) includes at least T1-weighted, T2-weighted, fluid-attenuated inversion recovery (FLAIR) and contrast-enhanced, T1-weighted sequences. The presumed site of origin is analyzed following a simplification of the concept of Patay et al., 2015 with the following levels: posterior/posterolateral brainstem, midline vermis or cerebellar hemispheres based on the preoperative imaging aspect.
The rate of agreement between both measures is reported.
Measured: From enrollment to the end of treatment at 6 weeks
Secondary outcomes (3)
The rate of agreement between the intraoperatively observed site of origin and the site of origin presumably associated with a molecular group (standard of reference) based on the developmental cell lineage concept of medulloblastoma
The intraoperatively observed site of origin is categorized into brainstem, cerebellar hemispheres and cerebellar vermis. The intraoperatively observed site of origin is assessed by an experienced pediatric neurosurgeon blinded to subgroup allocation and systemically documented in surgical forms.
Histopathological and genetic data are used to identify the molecular subgroup of the MB.
According to the current literature (Northcott et al., 2019), the site of origin presumably associated with the molecular group is categorized as brainstem for WNT-MB, the cerebellar hemisphere for SHH-MB and the cerebellar vermis for non-WNT/non-SHH-MB.
The rate of agreement between the intraoperatively observed site of origin (brainstem, cerebellar hemisphere, cerebellar vermis) and the site of origin presumably associated with a molecular group (brainstem, cerebellar hemisphere, cerebellar vermis) is reported for each molecular group separately.
Measured: From enrollment to the end of treatment at 6 weeks
The neurological outcome measured as KPS scores stratified by the intraoperatively observed site of origin
The intraoperatively observed site of origin is categorized into brainstem, cerebellar hemispheres and cerebellar vermis. The intraoperatively observed site of origin is assessed by an experienced pediatric neurosurgeon blinded to subgroup allocation and systemically documented in surgical forms.
The neurological outcome at 6 weeks- follow-up is assessed using the Karnofsy Performance Scale.
Patients are stratified by the intraoperatively observed site of origin into three groups (brainstem, cerebellar hemispheres, cerebellar vermis).
The KPS scores are reported as boxplots with median and interquartile range for the three groups separately.
Measured: From enrollment to the end of treatment at 6-weeks
Predictive accuracy of radiogenomic models
For construction of nomograms, the entire study sample will be split into a training cohort (TC) and a validation cohort (VC) in the ratio 7:3. Independent binary nomograms will be developed for each of the 4 subgroups. Receiver operating characteristics (ROC) curves will be generated following application of individual subgroup-specific nomograms to assign scores for patients in the TC. The methodology will be repeated in the VC. Optimal cutoff of total scores for individual subgroup-specific nomograms will be generated from the ROC curves from the TC for acceptable range of specificity and sensitivity, and their applicability was subsequently tested in the VC. Area under the curve (AUC) with 95% CI will be used for interpretation and reporting. MRI-based nomograms will be constructed two times, with the intraopely observed site of origin being the only additional feature. AUCs with 95% CI will be compared among the nomograms.
Measured: From enrollment to the end of treatment at 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2025
- Study start1 Mar 2024
- Primary completionMar 2026 (estimated)
- Study completionMar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MedulloblastomaStudies 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
- Prospective
- Enrolment
- 100 participants (estimated)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Christian Dorfer
- Sponsor type
- University, hospital or other
- Collaborators
- Medical University of Vienna, University Hospital Tuebingen, Utrecht University, Charite University, Berlin, Germany
- Responsible party
- Sponsor-investigator, Christian Dorfer, Medical University of Vienna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06814353
- Sponsor's protocol ID
- ISOP (EK 1967/2023)
- Acronym
- ISOP
- Status last verified
- Feb 2025
- First submitted
- 26 Dec 2024
- Last updated
- 7 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International Multicenter Prospective Cohort Study Investigating the Intraoperatively Observed Site of Origin and Growth Pattern of the Molecular Groups of Medulloblastoma
- ClinicalTrials.gov ID
- NCT06814353
- Acronym / sponsor ID
- ISOP / ISOP (EK 1967/2023)
- Conditions
- Medulloblastoma
- Keywords
- Medulloblastoma
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Christian DorferUniversity, hospital or other
- Collaborators
- Medical University of Vienna, University Hospital Tuebingen, Utrecht University, Charite University, Berlin, Germany
- Study start
- 1 Mar 2024 (actual)
- Primary completion
- Mar 2026 (estimated)
- Study completion
- Mar 2026 (estimated)
- First posted
- 7 Feb 2025 (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.
The goal of this observational study is to provide accurate and systematic data on the site of origin and growth pattern of medulloblastoma from a neurosurgical perspective. By integrating intraoperative, radiological and genetic classification data, this study will contribute to our current understanding of the development, site of origin and growth pattern of medulloblastoma and advance the predictive accuracy of radiogenomics models. Patients with histologically confirmed medulloblastoma who undergo surgical resection at a high-volume center with expertise in pediatric neurosurgery will be included.
The main questions it aims to answer are:
- Is there a significant difference between the intraoperatively observed site of origin and the preoperatively or postoperatively radiologically assessed site of origin of medulloblastoma?
- How does the intraoperatively observed site of origin align with the site of origin associated with the molecular group based on the developmental cell lineage concept of medulloblastoma?
- Does incorporating the intraoperatively observed site of origin as a feature improve the predictive accuracy of radiomic models for molecular group classification?
Participants will:
- Undergo intraoperative assessment of site of origin and growth pattern by an experienced pediatric neurosurgeon.
- Have their site of origin and growth pattern evaluated on pre- and postoperative magnetic resonance imaging by an neuroradiologist with expertise in pediatric brain tumors.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 1 Year
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| WNT-MB | – | Procedure: Intraoperative assessment of the site of origin and growth patternPatients with a medulloblastoma of the subgroup MB, WNT(Wingless)-activated according to the WHO classification 2021. |
| SHH-MB | – | Procedure: Intraoperative assessment of the site of origin and growth patternPatients with a medulloblastoma of the subgroup MB, SHH (Sonic Hedgehog)-activated according to the World Health Organization (WHO) classification 2021. This subgroup can be further differentiated into SHH-activated (SHH-MB) TP53-wildtype and MB, SHH-activated TP53-mutant. |
| Group 3 and Group 4 | – | Procedure: Intraoperative assessment of the site of origin and growth patternPatients with a medulloblastoma of the subgroup MB, non-WNT/non-SHH activated MB (Group 3 and 4) according to the WHO classification 2021. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Both adult and pediatric patients who undergo surgical resection of a histologically confirmed medulloblastoma at a center with expertise in pediatric neurosurgery
Key inclusion criteria (2 listed)
- Patients who are referred for surgical resection of a medulloblastoma at a hospital with expertise in pediatric neurooncology
- Both pediatric patients (aged < 18 years at the time of diagnosis) and adult patients (aged > 18 years at the time of diagnosis)
Key exclusion criteria (3 listed)
- Patients who are preoperatively admitted with severe tumor hemorrhage accompanied by clinical deterioration are excluded because these patients frequently do not receive magnetic resonance imaging as would be necessary for the present study, and the intraoperative assessment of the STO is limited in accuracy due to decreased visibility caused by the intra- and extratumoral hemorrhage.
- If an intraoperative complication occurs (e.g. bleeding) which impairs visibility of the neuroanatomic structures and does not allow an accurate assessment of the STO, the patient is excluded.
- If the histopathological and molecular analysis does not confirm the diagnosis of a MB, the patient has to be excluded as well.
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 |
|---|---|---|
| Intraoperative assessment of the site of origin and growth patternAlso: Assessment of the site and origin and growth pattern on preoperative MRI, Evaluation of anatomic features, Imaging feature extraction and characterization, Histopathological and molecular analysisThe assessment of the epicenter and extension and therefore the assumed origin of tumor growth is conducted by an experienced pediatric neurosurgeon blinded to group allocation based on the intraoperative impression. The anatomical features and site of origin are systematically documented on the surgical form in all cases of this study. | Procedure or surgery | Group 3 and Group 4, SHH-MB, WNT-MB |
Primary outcome
Rate of agreement between the intraoperatively observed site of origin and preoperatively radiologically assessed site of origin of medulloblastoma
Time frame: From enrollment to the end of treatment at 6 weeks
The intraoperatively observed site of origin is categorized into brainstem, cerebellar hemispheres and cerebellar vermis.The intraoperatively observed site of origin is assessed by an experienced pediatric neurosurgeon blinded to subgroup allocation and systemically documented in surgical forms. The location and site of origin is assessed by one expert neuroradiologist with expertise in pediatric brain tumor imaging who is blinded to the group allocation. Preoperative magnetic resonance imaging (MRI) includes at least T1-weighted, T2-weighted, fluid-attenuated inversion recovery (FLAIR) and contrast-enhanced, T1-weighted sequences. The presumed site of origin is analyzed following a simplification of the concept of Patay et al., 2015 with the following levels: posterior/posterolateral brainstem, midline vermis or cerebellar hemispheres based on the preoperative imaging aspect. The rate of agreement between both measures is reported.
3 secondary outcomes
The rate of agreement between the intraoperatively observed site of origin and the site of origin presumably associated with a molecular group (standard of reference) based on the developmental cell lineage concept of medulloblastoma
Time frame: From enrollment to the end of treatment at 6 weeks
The neurological outcome measured as KPS scores stratified by the intraoperatively observed site of origin
Time frame: From enrollment to the end of treatment at 6-weeks
Predictive accuracy of radiogenomic models
Time frame: From enrollment to the end of treatment at 6 weeks
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 7 Feb 2025.
- Individual participant dataSharing planned: Undecided
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Medulloblastoma, run by Christian Dorfer at 4 sites in 3 countries.
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 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: enrolling by invitation, as of the sponsor's last update on 7 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Germany, Austria, Netherlands. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of agreement between the intraoperatively observed site of origin and preoperatively radiologically assessed site of origin of medulloblastoma (time frame: From enrollment to the end of treatment at 6 weeks). 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 7 Feb 2025.