High-Risk NeuroblastomaPhase 2NCT07537400
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 study testing Naxitamab in people with High-Risk Neuroblastoma, run by Shaare Zedek Medical Center at 1 site in Israel.
Trial 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 more about this study
- Who is it for?
- Ages 12 months to 21 years
- Conditions the study is for
- High-Risk Neuroblastoma
- What is being tested?
- Naxitamab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteIsrael
- Study dates
- 23 Jun 2026to Apr 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
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- 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:
- 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.
- 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: -
- 1. Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Israel1 site, 1 recruiting
- Recruiting
Shaare Zedek Medical Center
Jerusalem
Rosi Goldenberg, 00972 524426504, rosig@szmc.org.il
Iris Fried, MD
About this study
Official title: 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
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.
- Conditions
- High-Risk Neuroblastoma
- Being tested
- Naxitamab
- Keywords
- Neuroblastoma, High-risk, Resistant to treatment, Newly-diagnosed, Naxitamab, Anti-GD2 antibody, Chemotherapy
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NaxitamabDrug
Also called Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)
Naxitamab and GM-CSF administered with COG type induction chemotherapy.
| Study group | Role | What they receive |
|---|---|---|
| Naxitamab and GM-CSF | Experimental | All enrolled patients receive Naxitamab and GM-CSF in combination with induction therapy for newly-diagnosed high-risk neuroblastoma Receives: Drug: Naxitamab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
Measured: 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
Measured: 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.
Measured: Post 2nd course, and post 5th course of induction therapy (therapy lasts approximately 4 and a half months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2026
- Study start23 Jun 2026
- Primary completionJun 2028 (estimated)
- Study completionApr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High-Risk NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shaare Zedek Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Responsible party
- Principal investigator, Iris Fried, Shaare Zedek Medical Center
- Investigators
- Iris Fried, MD, Principal investigator, Shaare Zedek Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07537400
- Sponsor's protocol ID
- 0234-25-SZMC
- Status last verified
- Sep 2026
- First submitted
- 9 Apr 2026
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified, anonymized data will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07537400
- Acronym / sponsor ID
- 0234-25-SZMC
- Conditions
- High-Risk Neuroblastoma
- Keywords
- Neuroblastoma, High-risk, Resistant to treatment, Newly-diagnosed, Naxitamab, Anti-GD2 antibody, Chemotherapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Shaare Zedek Medical CenterUniversity, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Study start
- 23 Jun 2026 (actual)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Apr 2032 (estimated)
- First posted
- 17 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Naxitamab and GM-CSF | Experimental | Drug: NaxitamabAll enrolled patients receive Naxitamab and GM-CSF in combination with induction therapy for newly-diagnosed high-risk neuroblastoma |
Who may qualify
- Ages
- 12 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (24 listed)
- Age - 12 months to 21 years at protocol enrollment
- Clinical eligibility criteria:
- Pathology:
- Molecular testing:
- Timing of patient enrollment
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NaxitamabAlso: Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)Naxitamab and GM-CSF administered with COG type induction chemotherapy. | Drug | Naxitamab and GM-CSF |
Primary outcome
Evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma
Time frame: Post 2nd course, and post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)
This measure will be assessed by evaluating treatment side effects.
Assess end-of-induction (EOI) response rates following concurrent Naxitamab and induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma.
Time frame: Post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)
This measure will be assessed using the 1993 International Neuroblastoma Response Criteria (INRC) criteria.
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)
2 secondary outcomes
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.
Time frame: Post 2nd course, and post 5th course of induction therapy (therapy lasts approximately 4 and a half months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: Yes. De-identified, anonymized data will be shared.
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Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Iris Fried, MD principal investigator
Shaare Zedek Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Naxitamab in people with High-Risk Neuroblastoma, run by Shaare Zedek Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 24 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dr. Iris Fried (00972 508573151, irisf@szmc.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma (time frame: Post 2nd course, and post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.