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Treatment for high-risk neuroblastoma using naxitamab and GM-CSF

A Pivotal Phase 2 Trial of Antibody Naxitamab (hu3F8) and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow

PHASE2 · Y-mAbs Therapeutics · NCT03363373

This study is testing a new treatment combining naxitamab and GM-CSF for children and adults with high-risk neuroblastoma who haven't responded well to previous therapies.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment122 (estimated)
Ages1 Year and up
SexAll
SponsorY-mAbs Therapeutics (industry)
Drugs / interventionschemotherapy, immunotherapy, naxitamab
Locations26 sites (Gainesville, Florida and 25 other locations)
Trial IDNCT03363373 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to treat children and adults with high-risk neuroblastoma who have primary refractory disease or an incomplete response to previous salvage treatments. Participants will receive naxitamab, a humanized monoclonal antibody targeting GD2, along with granulocyte-macrophage colony-stimulating factor (GM-CSF) over a treatment period of up to 101 weeks. The treatment regimen includes multiple cycles of GM-CSF and naxitamab, with patients monitored for up to five years after their first dose to assess long-term outcomes.

Who should consider this trial

Good fit: Ideal candidates include children and adults diagnosed with high-risk neuroblastoma who have not responded adequately to previous treatments.

Not a fit: Patients with neuroblastoma that has spread outside of bone and bone marrow or those with major organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve survival rates and outcomes for patients with high-risk neuroblastoma.

How similar studies have performed: Other studies using similar monoclonal antibody approaches have shown promise, indicating potential for success in this trial.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 response) evaluable in bone and/or bone marrow.
* Life expectancy ≥ 6 months

Exclusion Criteria:

* Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF
* Evaluable neuroblastoma outside bone and bone marrow
* Existing major organ dysfunction \> Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
* Active life-threatening infection

Where this trial is running

Gainesville, Florida and 25 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Neuroblastoma, Antibody, Neuroblastoma, Pediatric, Adult

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.