MULTISARC : MOLECULAR PROFILING OF ADVANCED SOFT-TISSUE SARCOMAS. A phase III study

简要总结

主要结局

次要结局

• Overall survival (OS): OS for the secondary objective is defined as the delay from the date of randomization to the date of death. OS for the assessment of efficacy of first-line treatment: OS will be defined as the delay from the date of onset of first-line treatment to the date of death. OS for the assessment of efficacy of targeted treatment: OS will be defined as the delay from the date of onset of targeted treatment to the date of death.
• Progression-free survival (PFS) for the secondary objective is defined as the delay from the date of randomization to the date of progression as per RECIST v1.1. or death, whichever occurs first. PFS for the assessment of efficacy of first-line treatment and the targeted treatment: PFS will be defined as the delay from the date of onset of first-line or targeted treatment to the date of progression as per RECIST v1.1. or death, whichever occurs first.
• Participants with targetable alteration(s) A participant will be considered as “presenting at least one targetable genomic alteration”, if the MTB consider that at least one genomic alteration identified can be matched with one of the drug available through the MULTISARC study.
• Best overall response for the assessment of efficacy of first-line treatment: Best response will be recorded from the date of onset of first-line treatment taking into account any requirement for confirmation as per RECIST 1.1 criteria. Best overall response under treatment for the assessment of efficacy of targeted treatment: Best response will be recorded from the date of onset of targeted treatment taking into account any requirement for confirmation as per RECIST 1.1 criteria.
• Objective response under treatment is defined as complete or partial response (CR, PR) as per RECIST v1.1 under first-line treatment. ). Following RECIST v1.1 recommendations, each participant will be assigned one of the following categories: complete response, partial response, stable disease, progression, invaluable for response. Moreover, claimed responses will have to be confirmed at least 4 weeks later to ensure responses identified are not the result of measurement errors.
• For the assessment of efficacy of first-line treatment and targeted treatment: Non-progression at 6 months is defined as complete or partial response (CR, PR) or stable disease (SD) at 6 months as per RECIST v1.1 under first-line or targeted treatment. Claimed responses will have to be confirmed at least 4 weeks later to ensure responses identified are not the result of measurement errors.
• Change in tumor size is defined as the difference (in percentage) in tumor size burden from the date of targeted treatment initiation (baseline) to the tumor assessment.
• Immunosequencing: Correlation of TCR-sequencing data with objective response (OR), progression-free survival (PFS) and Evaluate the association between immune profiles derived from ancillary analyses (VISIUM HD, multiplex IF, SomaScan proteomics) and treatment response, within an integrated immunosequencing framework. and overall survival (OS).
• Real cost of NGS: Micro-costing studies collect detailed data on resources utilized and the value of those resources. Cost of other resources: The French National Health Insurance reimburses the vast majority of other resources involved in the care of patients in this trial. These costs will be extracted from the French National Health Insurance data, and cost Per diem allowances: In case of sick leave.
• Utility data and Quality Adjusted Life Years Utility scores will be assessed through the EuroQol-5D-5L questionnaire. A Quality Adjusted Life Year is the survival (in years) weighted by a utility score.
• Incremental cost utility ratio: Our incremental cost-utility ratio is difference in cost between both strategies (NGS and no NGS) divided by the difference in QALYs.