Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5

NCT07537400 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 0234-25-SZMC
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This clinical trial will evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly-diagnosed patients with high-risk neuroblastoma. We aim to recruit 10 patients over the next 2 years.

Conditions

High-Risk Neuroblastoma

Keywords

Neuroblastoma; High-risk; Resistant to treatment; Newly-diagnosed; Naxitamab; Anti-GD2 antibody; Chemotherapy

Outcome Measures

Primary Outcomes (3)

• Evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma

  This measure will be assessed by evaluating treatment side effects.

  Post 2nd course, and post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)

• Assess end-of-induction (EOI) response rates following concurrent Naxitamab and induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma.

  This measure will be assessed using the 1993 International Neuroblastoma Response Criteria (INRC) criteria.

  Post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)

• Assess end-of-induction (EOI) response rates following additional cycles of Irinotecan-Temodar-Naxitamab-GM-CSF in patients with high risk neuroblastoma and less than partial response (PR) after induction with COG type chemotherapy and Naxitamab

  Post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)

Secondary Outcomes (2)

• Determine event-free survival (EFS) in newly diagnosed high-risk neuroblastoma patients

  From enrollment until completion of follow-up, 5 years from diagnosis

• Metastatic complete response after cycle 2 and at end of induction.

  Post 2nd course, and post 5th course of induction therapy (therapy lasts approximately 4 and a half months)

Study Arms (1)

Naxitamab and GM-CSF

EXPERIMENTAL

All enrolled patients receive Naxitamab and GM-CSF in combination with induction therapy for newly-diagnosed high-risk neuroblastoma

Drug: Naxitamab

Interventions

Naxitamab DRUG

Naxitamab and GM-CSF administered with COG type induction chemotherapy.

Also known as: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)

Naxitamab and GM-CSF

Eligibility Criteria

• Age: 12 Months - 21 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age - 12 months to 21 years at protocol enrollment
• Clinical eligibility criteria:
• Newly diagnosed high risk neuroblastoma.
• BOTH stage M (INRG - International Neuroblastoma Risk Group) and age ≥547 days.
• Patients ≥ 547 days of age who were initially diagnosed with INRG L1 or L2 disease but progress to Stage M without chemotherapy
• Patients \< 547 days of age with INRG Stage M or MS disease and patients of any age with INRG L2 with MYCN amplification (v-myc avian myelocytomatosis viral related oncogene)
• Pathology:
• Neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis, or
• demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
• Molecular testing:
• MYCN testing will be done by FISH (Fluorescence In Situ Hybridization) assessment of the FFPE (Formalin-Fixed Paraffin-Embedded) material submitted from the tumor mass. \> 4-fold increase in MYCN signals as compared to reference signals will qualify as MYCN amplified.
• ONCOMINE testing will evaluate ALK (Anaplastic Lymphoma Kinase) mutation
• Timing of patient enrollment
• Patients will be enrolled up to 6 weeks from primary diagnosis
• Pre-study imaging tests are acceptable up to 3 weeks prior to study enrollment and only if done after any pre-protocol chemotherapy.

You may not qualify if:

• Subjects who have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy.
• This will not restrict the emergency regimen at initial diagnosis.
• Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features.
• Patients ≥547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features.
• Patients with known bone marrow failure syndromes.
• Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable.
• Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy.
• Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential.
• Lactating females who plan to breastfeed their infants.
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation.

Sponsors & Collaborators

• Shaare Zedek Medical Center: lead
• Y-mAbs Therapeutics: collaborator

Study Sites (1)

Shaare Zedek Medical Center

Jerusalem, Israel

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

naxitamab; Colony-Stimulating Factors; Granulocyte-Macrophage Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Iris Fried, MD

  Shaare Zedek Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dr. Iris Fried

CONTACT

00972 508573151; irisf@szmc.org.il

Rosi Goldenberg

CONTACT

00972 524426504; rosig@szmc.org.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Pediatric Hemato-Oncology Unit

Study Record Dates

• First Submitted: April 9, 2026
• First Posted: April 17, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): April 1, 2032
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: SAP

Locations

IL (1)