NCT05489887

Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

A Phase II Study of Naxitamab Added to Induction Therapy for Subjects With Newly Diagnosed High-Risk Neuroblastoma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 93 (estimated)
• Sponsor: Giselle Sholler · Academic / Other
• Sex: All
• Age: 12 Months – 21 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. The initial chemotherapy will include 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.

Detailed description

This is a prospective, multicenter clinical trial in subjects with newly diagnosed high-risk neuroblastoma to evaluate the efficacy and safety of administering naxitamab with standard induction therapy. All subjects will be followed for disease response, event free survival, overall survival and toxicity. Extent of disease will be measured and assessed for changes throughout the course of the study. All efficacy analyses will be performed on the evaluable population which will consist of all enrolled subjects (subjects who initiate treatment with naxitamab in combination with GM-CSF plus standard induction therapy) and who have measurable disease at baseline. The initial chemotherapy Induction regimen will utilize sequential administration of 5 cycles of multi-agent chemotherapy. Naxitamab will be added to all 5 Induction cycles. Stem cell mobilization and collection will occur after the 2nd cycle of induction. Surgical resection of the primary tumor will ideally occur after the 4th cycle of Induction but may be delayed until after the 5th cycle of Induction if medically necessary. Disease status evaluations will occur at the following time points: (1) pre-treatment, (2) post Cycle 2 Induction (3) Prior to surgical resection (if performed), (4) End of Induction (which includes surgery and 5 cycles of chemotherapy), and (5) End of Additional/Salvage Therapy as needed. The current standard of care for high-risk neuroblastoma involves 5-7 cycles of induction chemotherapy with surgical removal of the tumor after 4-5 cycles of chemotherapy, followed by high-dose chemotherapy plus autologous stem cell transplant, then radiation to the primary tumor bed, followed by anti-GD2 immunotherapy and cis retinoic acid. This results in a less than 60% disease free survival for high-risk NB, a survival rate that still greatly needs improvement. Two areas in which improvements can be made include: 1) to improve response rate to induction chemotherapy and 2) to improve EFS by improving maintenance therapy to prevent relapse. We hypothesize that the addition of anti-GD2 therapy to induction chemotherapy will result in improved end of induction responses and improved survival.

Conditions

• Neuroblastoma

Interventions

Type	Name	Description
DRUG	Naxitamab	Naxitamab is a humanized (IgG1) anti-GD2 (hu3F8) monoclonal antibody for the treatment of neuroblastoma, osteosarcoma and other GD2-positive cancers. Naxitamab was granted accelerated approval by the FDA in 2020 as treatment (in combination with granulocyte-macrophage colony-stimulating factor - GM-CSF) for pediatric patients at least one year of age and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy

Timeline

• Start date: 2022-09-14
• Primary completion: 2029-09-01
• Completion: 2036-09-01
• First posted: 2022-08-05
• Last updated: 2026-02-03

Locations

23 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study