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

Starting soon · Phase 2

Conditions studied: High-Risk Neuroblastoma

In brief

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.

Key facts

Study ID

NCT07537400

Run by

Shaare Zedek Medical Center

People needed

10

Starts

2026-05-01

Expected to finish

2032-04-01

Last updated by the study team

2026-04-17

Who can join

Age: 1 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

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.
• Pre-treatment functional status:
• No active bacterial infection without adequate antibiotic therapy
• No uncontrolled viral infection - decision will be made after infectious disease consultation
• 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: -
• Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age,
• and SGPT (Serum Glutamic Pyruvic Transaminase - ALT) ≤ 10 x ULN* iii. Adequate cardiac function defined as: -
• Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.

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.

Where it is running

• Shaare Zedek Medical Center — Jerusalem, Israel

Full record on ClinicalTrials.gov

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