Phase 1 Study of Dinutuximab Beta with Chemotherapy for Children Ages 1 to 18 with High-Risk Neuroblastoma That Is Resistant or Has Returned After Standard Treatment

What this Trial Is About

This research investigates the safety and early effectiveness of dinutuximab beta combined with chemotherapy in children aged 1 to 18 years who have high-risk neuroblastoma that is resistant to standard treatments or has relapsed or progressed. Neuroblastoma is a common childhood cancer, especially under 5 years old, with different treatment approaches depending on risk. This trial focuses on patients for whom existing treatments have not led to lasting remission. Participants receive dinutuximab beta, an antibody targeting GD2 on neuroblastoma cells, given as a continuous infusion at 10 mgm2day for 5 days. This is combined with chemotherapy regimens including irinotecantemozolomide, topotecantemozolomide, or the N5N6 GPOH protocol. Treatment consists of 5 to 7 cycles, with the choice of chemotherapy based on prior treatments and tolerance. Two cycles of standard chemotherapy may be allowed before joining the study. During the study, patients undergo monitoring for side effects such as capillary leak syndrome, allergic reactions, and neurological or hematological toxicities for 12 months after treatment ends. Effectiveness is assessed by measuring response rates, progression-free survival, and event-free survival using imaging, tumor markers, and bone marrow exams. Additional studies on immunological and genetic factors are also explored. The trial plans to enroll 20 patients and assess safety and initial benefits.

Immunotherapy With Dinutuximab Beta in Combination With Chemotherapy for the Treatment of Patients With Primary Neuroblastoma Refractory to Standard Therapy and With Relapsed or Progressive Disease