Relapsed/refractory pediatric cancer, which was established by standard diagnostic methods OR initially diagnosed pediatric cancer patients for whom no standard treatment strategy is available or who have a dismal outcome with current treatment protocols.
Conditions
Interventions
none
Sponsors
Prinses Máxima Centrum voor kinderoncologie
Eligibility
Inclusion criteria
Inclusion criteria: • Relapsed/refractory pediatric cancer, which was established by standard diagnostic methods OR initially diagnosed pediatric cancer patients for whom no standard treatment strategy is available or who have a dismal outcome with current treatment protocols. In the case of very high risk primary disease, the results obtained in the frame of this study may be used at the physician’s discretion to improve a standard treatment strategy and may be applied a at later time point. • A tissue sample or tumor DNA/RNA as well as normal DNA was obtained as standard of care at diagnosis, resection or at the event of refractory or relapsed disease or is planned with the purpose to confirm the diagnosis or a suspected relapse/refractory disease. • Life-expectancy of at least 12 weeks. • Written informed consent according to local law and legislation • Age <30 years.
Exclusion criteria
Exclusion criteria: • Patients cannot participate in this study if there is no tissue or DNA/RNA available. • If patients and parents do not want to be informed about detected germline abnormalities
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To determine the objective response rates (complete response (CR), very good partial response (VGPR) or partial response (PR)) in pediatric patients with very high risk, relapsed or refractory tumors harboring actionable genomic alterations, treated with pathway-targeting agents compared to a non-treated cohort. | — |
Secondary
| Measure | Time frame |
|---|---|
| • To assess the number of patients in which the molecular tumor board can formulate a treatment advice based on the molecular profiling data. • To describe how many patients are treated according to this advice. (Note that the treatment intervention itself is not part of this protocol, but data will be captured.) • To determine the progression free survival in pediatric patients receiving targeted therapies for very high risk, relapsed or refractory tumors, compared to a non-treated cohort. • To determine the overall survival in pediatric patients receiving targeted therapies for very high risk, relapsed or refractory tumors, compared to a cohort treated with conventional non-targeted palliative therapy. • To describe the genomic changes as well as compound sensitivity in pediatric tumors, metastases and ctDNA between the time of initial diagnosis and progression or relapse, in cases for which paired tumor and/or plasma specimens are available. • To asses in how many patients sufficient material is available for organoid culture. • To asses in how many patients organoid culture is successful. • To asses in how many patients in vitro drug screening is successful. • To assess in how many patients additional potential effective compounds can be identified based on the drug sensitivity screening. • To determine the correlation between predictions based on the genomic profiling of patient-derived tumor biopsies and/or ctDNA and the results of the drug sensitivity screening. • To generate a standardized bioinformatics analysis tool to identify actionable events. • To implement the drug sensitivity results in the standard data analysis tool. • To assess the timeframe in which the molecular tumor board can formulate a treatment advice based on molecular profiling data alone and combined drug sensitivity screening results. • To assess how many patients are treated according to the in vitro drug sensitivity screening results. (Note that the treatment intervention itself is | — |
Contacts
Public ContactMiriam Stumpf
Prinses Máxima Centrum voor kinderoncologie
Outcome results
None listed