An observational study in Medulloblastoma, sponsored by Christian Dorfer. Enrolling by invitation at 4 sites in 3 countries. Per ClinicalTrials.gov, last updated 2025-02-07.
Sponsored by Christian Dorfer · Observational
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:
Participants will:
238 studies on the registry are indexed under Medulloblastoma; 51 are open to participants now.
This study's planned enrollment of 100 is below the median of 184 across 25 observational studies indexed under Medulloblastoma.
Browse Medulloblastoma studies →This is the only study on the registry with Christian Dorfer as lead sponsor.
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Both adult and pediatric patients who undergo surgical resection of a histologically confirmed medulloblastoma at a center with expertise in pediatric neurosurgery
Exclusion Criteria:
Patients with a medulloblastoma of the subgroup MB, WNT(Wingless)-activated according to the WHO classification 2021.
Procedure: Intraoperative assessment of the site of origin and growth pattern
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.
Procedure: Intraoperative assessment of the site of origin and growth pattern
Patients with a medulloblastoma of the subgroup MB, non-WNT/non-SHH activated MB (Group 3 and 4) according to the WHO classification 2021.
Procedure: Intraoperative assessment of the site of origin and growth pattern
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.
Also known as: 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
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.
Time frame: From enrollment to the end of treatment at 6 weeks
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.
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
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.
Time frame: 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.
Time frame: From enrollment to the end of treatment at 6 weeks
Plan to share: Undecided
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