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RecruitingNCT07537400Updated Sep 17, 2026

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

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

Phase
Phase 2
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
12 Months to 21 Years
Sex
All
01

Study 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.

Read the detailed description

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.

02

Conditions studied

  • High-Risk Neuroblastoma

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Keywords

  • Neuroblastoma
  • High-risk
  • Resistant to treatment
  • Newly-diagnosed
  • Naxitamab
  • Anti-GD2 antibody
  • Chemotherapy
03

Who can participate

Ages eligible
12 Months to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Age - 12 months to 21 years at protocol enrollment
  • Clinical eligibility criteria:

    1. Newly diagnosed high risk neuroblastoma.
    2. BOTH stage M (INRG - International Neuroblastoma Risk Group) and age ≥547 days.
    3. Patients ≥ 547 days of age who were initially diagnosed with INRG L1 or L2 disease but progress to Stage M without chemotherapy
    4. 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:

    1. Neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis, or
    2. demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
  • Molecular testing:

    1. 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.
    2. ONCOMINE testing will evaluate ALK (Anaplastic Lymphoma Kinase) mutation
  • Timing of patient enrollment

    1. Patients will be enrolled up to 6 weeks from primary diagnosis
    2. Pre-study imaging tests are acceptable up to 3 weeks prior to study enrollment and only if done after any pre-protocol chemotherapy.
  • Pre-treatment functional status:

    1. No active bacterial infection without adequate antibiotic therapy
    2. No uncontrolled viral infection - decision will be made after infectious disease consultation
    3. No uncontrolled organ dysfunction:

    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. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age,
  2. and SGPT (Serum Glutamic Pyruvic Transaminase - ALT) ≤ 10 x ULN* iii. Adequate cardiac function defined as: -

1. Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.

Exclusion criteria

Exclusion Criteria:

  • 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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (estimated)

Study arms

  • Experimental
    Naxitamab and GM-CSF

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

    Drug: Naxitamab

Interventions

  • DrugNaxitamab

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

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

05

What researchers measure

Primary outcomes

  1. 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)

  2. 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)

  3. 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)

Secondary outcomes

  1. 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

  2. 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)

06

Study locations

1 of 1 sites recruiting
  • Shaare Zedek Medical Center
    Jerusalem, Israel
    • Rosi Goldenberg · Contact · rosig@szmc.org.il · 00972 524426504
    • Iris Fried, MD · Principal investigator
    Recruiting
07

References and documents

Individual participant data

Plan to share: Yes — De-identified, anonymized data will be shared.

Supporting information: Sap

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT07537400
Lead sponsor
Shaare Zedek Medical Center
Collaborators
Y-mAbs Therapeutics
Responsible party
Iris Fried (Director of the Pediatric Hemato-Oncology Unit, Shaare Zedek Medical Center) — Principal investigator
First posted
Apr 17, 2026
Start date
Jun 23, 2026
Primary completion
Jun 2028 (estimated)
Completion
Apr 2032 (estimated)
Last update
Sep 17, 2026

Study contacts

Dr. Iris Fried
Contact
irisf@szmc.org.il
00972 508573151
Rosi Goldenberg
Contact
rosig@szmc.org.il
00972 524426504
Iris Fried, MD
principal investigator · Shaare Zedek Medical Center

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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