ClinicalTrials.gov record NCT07537400

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Phase II Trial of Naxitamab, Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Combination With Induction Chemotherapy for Patients With Newly-Diagnosed High-Risk Neuroblastoma

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age1 year old to 21 years old
SexAll
LocationIsrael
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This phase 2 study is for children and young adults (12 months to 21 years) with newly diagnosed high‑risk neuroblastoma. Participants receive Children's Oncology Group (COG)‑type induction chemotherapy with Naxitamab and GM‑CSF added during induction cycles 1–5. The study measures safety, response during induction, and longer-term outcomes.

What it is studying. The study tests the safety of combining Naxitamab and granulocyte‑macrophage colony stimulating factor (GM‑CSF) with standard COG‑type induction chemotherapy. It also measures tumor response after two cycles and at the end of induction, evaluates response after additional irinotecan‑temodar‑naxitamab‑GM‑CSF in patients with less than a partial response, and follows event‑free survival over time.

What the researchers hope to learn. Scientific goals: 1) Evaluate safety of chemoimmunotherapy with Naxitamab and COG‑type induction chemotherapy by tracking treatment side effects (assessed post‑cycle 2 and post‑cycle 5). 2) Measure end‑of‑induction (EOI) response rates using the 1993 International Neuroblastoma Response Criteria (INRC). 3) Assess response after additional irinotecan‑temodar‑naxitamab‑GM‑CSF in patients with less than partial response after induction. 4) Determine event‑free survival (EFS) and assess metastatic complete response after cycle 2 and at EOI.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Naxitamab

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive COG‑type induction chemotherapy with Naxitamab and GM‑CSF added to induction cycles 1–5. Complete assessments are done before enrollment, after cycle 2, and at the end of induction; these assessments include imaging (examples listed in the record: MIBG, CT, MRI, or PET‑DOPA) and may include bone marrow testing and surgery when clinically needed. The registry gives conflicting information about what happens after induction if the disease has less than a partial response: the study description says these patients are taken off the protocol, while one of the study’s outcome measures describes additional cycles of irinotecan–temodar–naxitamab–GM‑CSF for patients with less than a partial response after induction. Ask the study team which applies. A patient diagnosed with progressive disease at any stage is taken off the protocol. Follow‑up assessments occur after surgery and then every 4 months for 5 years from diagnosis; consolidation, radiation, and post‑consolidation treatments are provided at the treating physician's discretion.

How the study is structured. Phase 2, single‑group interventional trial with no masking. All enrolled patients receive Naxitamab and GM‑CSF together with COG‑type induction chemotherapy for induction cycles 1–5. Assessments are done before enrollment, after the second cycle, and at the end of induction; patients with less than a partial response or with progressive disease are removed from the protocol. Further consolidation, radiation, and post‑consolidation therapy are given at the treating physician's discretion. Follow‑up assessments occur after surgery and every 4 months for 5 years from diagnosis.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry lists this study under High-Risk Neuroblastoma. Does it apply to my diagnosis?
  • The listed sites are in Israel. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Gaucher disease

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Important dates

Study start
Jun 23, 2026
Primary completion
June 2028 (estimated)
Study completion
April 2032 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Sep 17, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Shaare Zedek Medical Center
Principal investigator
Iris Fried, Director of the Pediatric Hemato-Oncology Unit
Shaare Zedek Medical Center
Collaborators
  • Y-mAbs Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
eligibility criteria
The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
immunosuppressive
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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