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GM-CSF+ Naxitamab

Phase 2

Neuroblastoma | Monoclonal antibody | Oncology |Y-mAbs Therapeutics, Inc.|Last Updated: Feb 19, 2026

Target and mechanism

ModalityMonoclonal antibody

Also known as GM-CSF

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment122

FDA Designations

No designations recorded

Clinical trial landscape

GM-CSF+ Naxitamab · 1 trial · 1 indication

Phase 2 1
NCT03363373Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone MarrowNeuroblastoma
RECRUITING122 Analytics
PHASE2RECRUITING
Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
NeuroblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Response rate during Naxitamab treatment
101 weeks

Overall objective response rate (ORR) during the Naxitamab treatment period that will be centrally assessed according to the International Neuroblastoma Response Criteria (INRC) modified with 123I-MIBG criteria and following the use of 18F FDG-PET for MIBG non-avid lesions.

Secondary Endpoints

Incidence of adverse events and serious adverse events
101 weeks
Duration of Response (DoR)
101 weeks
Complete Response Rate
101 weeks
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GM-CSF + NaxitamabEXPERIMENTALEach investigational cycle is started with 5 days of GM-CSF administered at 250 µg/m2/day in advance of the start of Naxitamab administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, Naxitamab is administered at 3 mg/kg/day on days 1, 3, and 5 totalling 9 mg/kg per cycle. Treatment cycles are repeated every 4 weeks until CR or PR followed by 5 additional cycles every 4 weeks (±1 week). Subsequent cycles are repeated every 8 weeks (±2 weeks) through 101 weeks from first infusion at the discretion of the investigator. After end of treatment patients will enter a long-term follow up for up to 3 years after end of treatment visit.

Interventions

NameTypeDescription
GM-CSF + NaxitamabBIOLOGICALGranulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody
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Eligibility Criteria

Age Range1 Year to N/A
SexALL
Healthy VolunteersNo
Study Sites26

Inclusion Criteria: * Diagnosis of neuroblastoma as defined per International Neuroblastoma Response Criteria * High-risk neuroblastoma patients with either primary refractory disease or incomplete response to salvage treatment (in both cases including stable disease, minor response and partial res...

Countries:United StatesCanadaDenmarkFranceGermanyHong KongIndiaItalySpainUnited Kingdom
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Frequently asked questions about GM-CSF+ Naxitamab

What is GM-CSF+ Naxitamab used for?

GM-CSF+ Naxitamab is used for the treatment of neuroblastoma, specifically in high-risk patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow. It is an investigational combination therapy being studied in a Phase 2 clinical trial.

What does GM-CSF+ Naxitamab target?

The drug is a monoclonal antibody-based therapy. Naxitamab is a monoclonal antibody that targets the GD2 antigen, which is expressed on neuroblastoma cells. GM-CSF is included to stimulate the immune system. The combination is designed to enhance the immune response against the cancer.

Who makes GM-CSF+ Naxitamab?

GM-CSF+ Naxitamab is being developed by Y-mAbs Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol YMAB on the NASDAQ exchange.

What phase is GM-CSF+ Naxitamab in?

GM-CSF+ Naxitamab is currently in Phase 2 clinical development. It is an investigational therapy, meaning it has not yet been approved by regulatory authorities such as the FDA. The ongoing Phase 2 trial is actively recruiting participants.

What clinical trials is GM-CSF+ Naxitamab in?

GM-CSF+ Naxitamab is being studied in a single Phase 2 clinical trial with the identifier NCT03363373. The trial is titled 'Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow' and is enrolling 122 patients across multiple countries.

Is GM-CSF+ Naxitamab the same as GM-CSF?

GM-CSF+ Naxitamab is a combination therapy that includes GM-CSF (granulocyte-macrophage colony-stimulating factor) and naxitamab. While GM-CSF is a component, the drug as a whole is distinct from GM-CSF alone. The combination is being studied specifically for neuroblastoma.