Refractory primary bone tumors
Conditions
Brief summary
EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm., Determination of the dose of the test substance in patients between 9 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved., To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance., Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG.
Detailed description
PFS (Progression-Free Survival) - progression-free survival - will be measured from randomization to finding disease progression in imaging studies., OS (Overall Survival) - will be measured from randomization to death due to neoplastic disease., ORR (Overall Response Rate) - Percentage of patients who achieved a protocol-defined response to treatment., Time to reach the target serum concentration of the test substance., Maximum serum concentration at steady state Cmaxs., Steady-state trough serum concentration Cminss., Casual steady-state serum concentration Css., Exposure to Ctau., Time to steady-state concentration of the test substance in the serum.
Interventions
DRUGREGORAFENIB
Sponsors
Instytut Matki I Dziecka
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm., Determination of the dose of the test substance in patients between 9 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved., To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance., Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG. | — |
Secondary
| Measure | Time frame |
|---|---|
| PFS (Progression-Free Survival) - progression-free survival - will be measured from randomization to finding disease progression in imaging studies., OS (Overall Survival) - will be measured from randomization to death due to neoplastic disease., ORR (Overall Response Rate) - Percentage of patients who achieved a protocol-defined response to treatment., Time to reach the target serum concentration of the test substance., Maximum serum concentration at steady state Cmaxs., Steady-state trough serum concentration Cminss., Casual steady-state serum concentration Css., Exposure to Ctau., Time to steady-state concentration of the test substance in the serum. | — |
Countries
Poland
Outcome results
None listed