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Naxitamab

Phase 2

High-Risk Neuroblastoma | Small molecule | Oncology |Y-mAbs Therapeutics, Inc.|Last Updated: Apr 17, 2026

Success Probability

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment10

FDA Designations

No designations recorded

Clinical trial landscape

Naxitamab · 3 trials · 5 indications

Phase 2 2Phase 1 1
NCT07537400Patients 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 1-5High-Risk Neuroblastoma
NOT YET_RECRUITING10 Analytics
NCT05489887Naxitamab Added to Induction for Newly Diagnosed High-Risk NeuroblastomaNeuroblastoma
RECRUITING93 Analytics
PHASE2NOT YET_RECRUITING
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
High-Risk NeuroblastomaUnlock trial analytics
PHASE2RECRUITING
Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
NeuroblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly diagnosed patients with high-risk neuroblastoma
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.
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
Post 5th course of chemoimmunotherapy (therapy lasts approximately 4 and a half months)
Number of participants with Complete Response (CR) rate per 1993 INRC guidelines
6 to 12 months

Measured by the presence of radiologically assessable disease by cross-sectional computed tomography (CT) or Magnetic resonance imaging (MRI) imaging and/or by metaiodobenzylguanidine (MIBG) or positron emission tomography (PET) scans and bone marrow (BM) response for subjects with newly-diagnosed high-risk neuroblastoma according to the 1993 International Neuroblastoma Response Criteria (INRC) and compare to relevant historical controls.

Recommended duration of naxitamab infusion
Assessed at the end of the first cycle of treatment, where 1 cycle is 21 days

The duration of naxitamab infusion that is safe and tolerable will be measured using the endpoint of an occurrence of any dose limiting toxicity. A modified 3+3 design will be used, where the 'modification' permits a single duration level de-escalation in addition to the typical duration group level escalation.

Secondary Endpoints

Determine event-free survival (EFS) in newly diagnosed high-risk neuroblastoma patients
From enrollment until completion of follow-up, 5 years from diagnosis
Metastatic complete response after cycle 2 and at end of induction.
Post 2nd course, and post 5th course of induction therapy (therapy lasts approximately 4 and a half months)
Number of participants with CR + Very Good Partial Response (VGPR) rate and objective response rate (ORR; CR + VGPR + PR)
6 to 12 months
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Naxitamab and GM-CSFEXPERIMENTALAll enrolled patients receive Naxitamab and GM-CSF in combination with induction therapy for newly-diagnosed high-risk neuroblastoma
HRNB Newly diagnosed subjectsEXPERIMENTAL5 cycles of standard of care induction + naxitimab Naxitimab on Days 1, 3, and 5 of each cycle
Naxitamab Infusion+ Ironotecan + Temozolomide + SargramostimEXPERIMENTALParticipants will be enrolled in a modified 3+3 design to establish a maximum tolerated, infusion duration for Naxitamab. Dosage will start at Duration Level 1 and will de-escalate to Duration Level -1 or escalate to Duration Level 2 according to dose-limiting toxicity (DLT) criteria. * Baseline visit * Cycle 1 (21 day cycle): * Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily * Days 1 through 5: Predetermined dose of Temozolomide 1x daily * Days 1 through 5: Predetermined dose of Ironotecan 1x daily * Days 6 through 12: predetermined dose of Sargramostim 1x daily * Cycles 2 through 6 (21 day cycles): * Imaging on Cycles 2, 4, and 6 * Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily * Days 1 through 5: Predetermined dose of Temozolomide 1x daily * Days 1 through 5: Predetermined dose of Ironotecan 1x daily * Days 6 through 12: predetermined dose of Sargramostim 1x daily * End of treatment visit * Follow up at 1 year post-treatment

Interventions

NameTypeDescription
NaxitamabDRUGNaxitamab and GM-CSF administered with COG type induction chemotherapy.
IrinotecanDRUGTopoisomerase Inhibitor, single-dose vial, via intravenous infusion per standard of care
TemozolomideDRUGAlkylating agent, capsule, via orally (by mouth) per standard of care
Sargramostim granulocyte-macrophage colony stimulating factorDRUGRecombinant human granulocyte-macrophage colony stimulating factor (rhu GM-CSF), multi-use vial, via subcutaneously (under the skin) injection per standard of care
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Eligibility Criteria

Age Range12 Months to 21 Years
SexALL
Healthy VolunteersNo
Study Sites1

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 ...

Countries:IsraelUnited StatesCanada
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Frequently asked questions about Naxitamab

What is Naxitamab used for?

Naxitamab is an investigational antibody being studied for the treatment of neuroblastoma, including high-risk neuroblastoma. It is being evaluated in patients with newly diagnosed high-risk disease and in those with relapsed or refractory neuroblastoma. The drug is currently in clinical development and has not been approved by the FDA.

What does Naxitamab target?

Naxitamab is a monoclonal antibody, indicated by its -mab suffix. Its specific molecular target has not been disclosed in available clinical trial information. The drug is being studied for its potential role in treating neuroblastoma, a cancer that primarily affects children.

Who is developing Naxitamab?

Naxitamab is being developed by Y-mAbs Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol YMAB. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with neuroblastoma.

What phase is Naxitamab in?

Naxitamab is in Phase 2 clinical development. It is being studied in multiple ongoing trials, including a Phase 2 trial for newly diagnosed high-risk neuroblastoma and a Phase 1 trial for relapsed or refractory neuroblastoma. The drug remains investigational and is not yet approved.

What clinical trials is Naxitamab in?

Naxitamab is being studied in several recruiting trials. NCT05489887 is a Phase 2 trial adding naxitamab to induction therapy for newly diagnosed high-risk neuroblastoma. NCT07027748 is a Phase 1 feasibility study of prolonged naxitamab with irinotecan and temozolomide for relapsed or refractory neuroblastoma. NCT07537400 is a Phase 2 trial in Israel for newly diagnosed high-risk neuroblastoma.

Is Naxitamab the same as other neuroblastoma treatments?

Naxitamab is a distinct investigational antibody being developed specifically for neuroblastoma. It is being tested in combination with other agents such as irinotecan, temozolomide, and GM-CSF in clinical trials. No alternative names for naxitamab have been reported in the trial information.