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Study 201

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

Phase 2N = 74NCT03363373

Population
Patients with relapsed or refractory high-risk neuroblastoma and residual disease in bone or bone marrow; 74 patients treated, efficacy population 52.
Design
Single-arm, non-randomised phase 2 trial of naxitamab (3 mg/kg/day on days 1, 3 and 5) with GM-CSF every 4 weeks until complete or partial response, then five further cycles. The paper reports a prespecified interim analysis (enrolment April 2018 to December 2021, data cutoff 31 December 2021).
Primary endpoint
ORR
Arms
  • Naxitamab plus GM-CSF (experimental, n = 52): Naxitamab 3 mg/kg/day on days 1, 3 and 5 with GM-CSF (days -4 to 5) every 4 weeks
People
Mora J, senior author Kushner BH
Show the numbers
Results reported for Study 201
EndpointAnalysisNaxitamab plus GM-CSFControlEffect
ORREfficacy population (52 of 74 treated)Overall response (complete or partial response) by the 2017 International Neuroblastoma Response CriteriainterimMora 202550% (36–64%)—26 responders: complete response 38% and partial response 12%. This interim analysis postdates the FDA approval (25 November 2020); it is not the data set the FDA reviewed.
PFSEfficacy population (52 of 74 treated)interimMora 202535% at 12 mo (16–54%)—Secondary endpoint.
OSEfficacy population (52 of 74 treated)interimMora 202593% at 12 mo (80–98%)—Secondary endpoint.
Results reported for Study 201
EndpointAnalysisNaxitamab plus GM-CSFControlEffect
ORREfficacy population (52 of 74 treated)Overall response (complete or partial response) by the 2017 International Neuroblastoma Response CriteriainterimMora 202550% (36–64%)—26 responders: complete response 38% and partial response 12%. This interim analysis postdates the FDA approval (25 November 2020); it is not the data set the FDA reviewed.
PFSEfficacy population (52 of 74 treated)interimMora 202535% at 12 mo (16–54%)—Secondary endpoint.
OSEfficacy population (52 of 74 treated)interimMora 202593% at 12 mo (80–98%)—Secondary endpoint.
Primary publication: Mora J, Chan GCF, Morgenstern DA, Amoroso L, Nysom K, Faber J, et al. The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial. Nat Commun. 2025;16(1):1636. PubMedDOI

Appears in

Sources

  1. Mora J, Chan GCF, Morgenstern DA, Amoroso L, Nysom K, Faber J, et al. The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial. Nat Commun. 2025;16(1):1636.

    PubMedDOI