Phase III Study of Pucotenlimab with Standard Chemotherapy for Children and Adolescents With Intermediate to High-Risk Rhabdomyosarcoma as Pre-Surgery Treatment

What this Trial Is About

Researchers are conducting a Phase III clinical trial to study children and adolescents aged 1 to 18 years with intermediate to high-risk rhabdomyosarcoma, a type of pediatric cancer. The study aims to evaluate the safety, tolerability, and pharmacokinetics of Pucotenlimab combined with the standard chemotherapy regimen. This research focuses on how well this combination works as neoadjuvant therapy before surgery and how safe it is for young patients. In Phase I, the study uses a dose escalation design with three Pucotenlimab dose levels 1 mgkg, 3 mgkg, and 6 mgkg given every three weeks alongside standard chemotherapy. In Phase II, after identifying the recommended dose, all participants receive Pucotenlimab combined with the standard chemotherapy every three weeks for 2 to 4 cycles before surgery. The standard chemotherapy regimen follows the SYSUCC-RMS-2017 protocol used in China. During the study, participants will have regular assessments including monitoring for dose-limiting toxicities within 28 days of dosing and evaluation of the pathological complete response from treatment to surgery. Researchers will closely monitor safety, treatment effects, and clinical visits with laboratory tests and imaging as needed. The total participation period includes treatment cycles followed by surgery, ending by December 2026.

Effectiveness of Pucotenlimab Combined With Standard Chemotherapy Regimen