neuroblastoma a rare childhood cancer of the nerve cells
Conditions
Interventions
No medicinal study intervention is intended with this protocol. Patients will have standard treatments for their disease. Half of the patients will be randomised to have monthly blood samples taken to
Sponsors
Prinses Máxima Centrum voor Kinderoncologie
Eligibility
Age
No minimum to 64 Years
Inclusion criteria
Inclusion criteria: - Initial diagnosis of high-risk neuroblastoma as per SIOPEN modified INRG staging system2 defined as: · Stage M neuroblastoma above 365 days of age at diagnosis and stage Ms neuroblastoma 12-18 months old, any MYCN status · Stage M, Ms and L2 neuroblastoma any age with MYCN Amplification or focal high level MYC of MYCL amplification - From 6 months of age, to allow for monthly sampling - After initiation of second-line (or third- or fourth-line) therapy for relapsed or refractory disease - Most recent evaluations after therapy for event shows complete remission (CR), partial response (PR), mixed response (MR) or stable disease (SD) for at least 2 months as per local disease assessment - Written informed consent of both legal representatives
Exclusion criteria
Exclusion criteria: Participation in an experimental therapeutic clinical trial, where MONALISA trial randomisation methodology would impact clinical trial assessments of the therapeutic trial. An experimental therapeutic trial is defined as a therapeutic trial where the patient is receiving treatment in the experimental treatment phase of the trial. Patients continuing treatment off experimental trial or on maintenance therapy post-trial are eligible for inclusion for the MONALISA trial when they meet all other criteria. Additionally, patients treated according to local treatment protocol are also eligible for inclusion in the MONALISA trial.More than 3 months after ending relapse/refractory event therapy. This is to prevent bias in time to event as the likelihood of a new event increases over time.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to molecular event detection with liquid biopsies compared to clinical event detection with standard of care disease assessment in patients with relapsed neuroblastoma that have achieved a response or stable disease to prior treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Overall survival2. The positive and negative predictive values of liquid biopsies to detect an event3. The patient and clinician evaluation of the use of liquid biopsies for early event detection4. Change in care, including frequency of disease assessment, starting new treatment or a change in treatment regimen initiated by a liquid biopsy result 5. The identification of druggable targets, for example Anaplastic Lymphoma Kinase (ALK) mutations, the proportion of patients with druggable targets identified and the initiation of targeted therapy6. Detection of HR genetic aberrations in liquid biopsies, such as segmental chromosomal aberrations and MYCN Amplification 7. Compliance of liquid biopsy sampling frequency and patient reported evaluation of monthly liquid biopsies 8. Turnaround time of clinical reports from multiple reference labs and the capacity to perform clinical liquid biopsies testing conform local regulations9. Retrospective analysis of the predictive value of different technical and clinical cut off values in liquid biopsy samples from MONALISA trial 10. Observer Reported Outcome (ObsROs) and Patient Reported Outcomes (PROs)11. Completion rate of ObsRO and PRO assessments over time and caregiver evaluation of feasibility and usability of HRQoL assessment12. Evaluating socioeconomic risk factors, assessed by the psychosocial assessment tool, across patients eligible for MONALISA during the pre-screening phase and compare these with rates of inclusion in this trial 13. To assess if psychosocial and socioeconomic factors predict child and parent outcomes.14. Completion percentage of weekly symptom monitoring via a mobile device application and caregiver evaluation of feasibility and usability of weekly symptom screening 15. Quantification of weekly symptoms, possible changes in weekly symptoms over time and differences in weekly symptoms between arm A and arm B. | — |
Countries
Austria, Belgium, Czech Republic, France, Germany, Greece, Italy, Netherlands, Spain, United Kingdom
Contacts
Public ContactG.A.M Tytgat
Prinses Máxima Centrum voor Kinderoncologie
Outcome results
None listed