A Phase 2 interventional study of Naxitamab in High-Risk Neuroblastoma, sponsored by Shaare Zedek Medical Center. Recruiting at 1 site in Israel. Open to participants aged 12 Months to 21 Years. Per ClinicalTrials.gov, last updated 2026-09-17.
Sponsored by Shaare Zedek Medical Center · Phase 2, Interventional, and Treatment
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.
This clinical trial will use the backbone of the St. Jude NB2012 protocol which assessed the hu14.18K322A anti-GD2 antibody with COG type induction. The current study will replace the hu14.18K322A with Naxitamab. Naxitamab is an anti-GD2 antibody that was evaluated as part of multiple chemoimmunotherapy protocols with favorable side effect profile and proven efficacy.
Patients will receive COG type recommended therapy as administered on ANBL1531 and NB2012 protocols with the addition of Naxitamab and GM-CSF to Induction Cycles 1-5. Further treatment, including: Consolidation, Radiation and Post-Consolidation therapy will be given at the discretion of the treating physician.
Patients will undergo complete assessment prior to trial enrollment, after 2 cycles, and post chemotherapy to allow for accurate assessment of response to treatment. If less than partial response, the patient will be taken off the protocol. A patient diagnosed with progressive disease at any stage of treatment will be taken off the protocol. Follow-up assessments will be carried out post surgery, and every 4 months for 5 years from diagnosis.
Clinical eligibility criteria:
Pathology:
Molecular testing:
Timing of patient enrollment
Pre-treatment functional status:
i. Renal function based on the Schwartz formula (Schwartz et al. J. Peds, 106:522, 1985). If creatinine level is abnormal chemotherapy treatment will be planned in consultation with Nephrology service. Naxitamab will be initiated if creatinine level is up to 1.2 of normal.
ii. Adequate liver function defined as: -
1. Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.
Exclusion Criteria:
All enrolled patients receive Naxitamab and GM-CSF in combination with induction therapy for newly-diagnosed high-risk neuroblastoma
Drug: Naxitamab
Naxitamab and GM-CSF administered with COG type induction chemotherapy.
Also known as: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)
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.
Time frame: 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.
Time frame: 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
Time frame: Post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)
Determine event-free survival (EFS) in newly diagnosed high-risk neuroblastoma patients
Time frame: From enrollment until completion of follow-up, 5 years from diagnosis
Metastatic complete response after cycle 2 and at end of induction.
To be assessed using one of the following: MIBG - Metaiodobenzylguanidin; CT - Computed Tomography; MRI - Magnetic Resonance Imaging; PET-DOPA - Fluorine-18 Fluorodopa Positron Emission Tomography.
Time frame: Post 2nd course, and post 5th course of induction therapy (therapy lasts approximately 4 and a half months)
Plan to share: Yes — De-identified, anonymized data will be shared.
Supporting information: Sap
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Shaare Zedek Medical Center