A randomized phase II study comparing the safety and efficacy of mifamurtide and standard therapy containing sorafenib in pediatric and young adult patients with high-risk osteosarcoma

Condition studied

Relapsed/refractory osteosarcoma, High-grade osteosarcoma

Investigational medicinal product(s)

Eligibility

Primary endpoint

EFS (Event-Free Survival) – the time from randomization to the first event, i.e., death, disease progression, or disease relapse, whichever occurs first. Assessment will be conducted from the date of randomization to the date of the event or to the date of the last available assessment.

Endpoint detail

OS (Overall Survival) – Overall survival will be measured from randomization to death from any cause; for patients alive at the end of follow-up, data will be censored at the date of last confirmed contact., PFS (Progression-Free Survival) – Progression-free survival will be measured from randomization to the date of disease progression or death, whichever occurs first., ORR (Overall Response Rate) – Response rate, defined as the percentage of patients with the best documented complete response (CR) or partial response (PR), assessed according to RECIST v1.1 at the time points specified in the imaging schedule., Evaluation of the safety of mifamurtide in patients with osteosarcoma. Safety will be assessed based on the number of serious adverse events (SAEs), the number of adverse events (AEs), a physical examination with analysis of recorded vital signs and laboratory abnormalities according to NCI CTCAE v5.0, from the start of treatment to the end of safety follow-up according to the study schedule.