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The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial

Author

Listed:
  • Jaume Mora

    (Hospital Sant Joan de Déu)

  • Godfrey C. F. Chan

    (Queen Mary Hospital & Hong Kong Children’s Hospital
    The University of Hong Kong)

  • Daniel A. Morgenstern

    (University of Toronto)

  • Loredana Amoroso

    (IRCCS Istituto Giannina Gaslini
    University of Rome)

  • Karsten Nysom

    (Copenhagen University Hospital – Rigshospitalet)

  • Jörg Faber

    (University Medical Center of the Johannes Gutenberg University Mainz)

  • Arthur Wingerter

    (University Medical Center of the Johannes Gutenberg University Mainz)

  • Melissa K. Bear

    (Riley Hospital for Children)

  • Alba Rubio-San-Simon

    (Hospital Infantil Universitario Niño Jesús)

  • Blanca Martínez de Las Heras

    (Hospital Universitario y Politecnico La Fe)

  • Karen Tornøe

    (Y-mAbs A/S)

  • Maria Düring

    (Y-mAbs A/S)

  • Brian H. Kushner

    (Memorial Sloan Kettering Cancer Center)

Abstract

In this single-arm, non-randomized, phase 2 trial (NCT03363373), 74 patients with relapsed/refractory high-risk neuroblastoma and residual disease in bone/bone marrow (BM) received naxitamab on Days 1, 3, and 5 (3 mg/kg/day) with granulocyte-macrophage colony-stimulating factor (Days -4 to 5) every 4 weeks, until complete response (CR) or partial response (PR) followed by 5 additional cycles every 4 weeks. Primary endpoint in the prespecified interim analysis was overall response (2017 International Neuroblastoma Response Criteria). Among 26 responders (CR + PR) in the efficacy population (N = 52), 58% had refractory disease, and 42% had relapsed disease. Overall response rate (ORR) was 50% (95% CI: 36-64%), and CR and PR were observed in 38% and 12%, respectively. With the 95% CI lower limit for ORR exceeding 20%, the primary endpoint of overall response was met. Patients with evaluable bone disease had a 58% (29/50) bone compartment response (CR, 40%; PR, 18%). BM compartment response was 74% (17/23; CR, 74%). One-year overall survival and progression-free survival (secondary endpoints) were 93% (95% CI: 80-98%) and 35% (95% CI: 16-54%), respectively. Naxitamab-related Grade 3 adverse events included hypotension (58%) and pain (54%). Overall, naxitamab demonstrated clinically meaningful efficacy with manageable safety in patients with residual neuroblastoma in bone/BM.

Suggested Citation

  • Jaume Mora & Godfrey C. F. Chan & Daniel A. Morgenstern & Loredana Amoroso & Karsten Nysom & Jörg Faber & Arthur Wingerter & Melissa K. Bear & Alba Rubio-San-Simon & Blanca Martínez de Las Heras & Kar, 2025. "The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial," Nature Communications, Nature, vol. 16(1), pages 1-13, December.
    • Handle: RePEc:nat:natcom:v:16:y:2025:i:1:d:10.1038_s41467-025-56619-x
    DOI: 10.1038/s41467-025-56619-x
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    References listed on IDEAS

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    1. Karin Schmelz & Joern Toedling & Matt Huska & Maja C. Cwikla & Louisa-Marie Kruetzfeldt & Jutta Proba & Peter F. Ambros & Inge M. Ambros & Sengül Boral & Marco Lodrini & Celine Y. Chen & Martin Burker, 2021. "Spatial and temporal intratumour heterogeneity has potential consequences for single biopsy-based neuroblastoma treatment decisions," Nature Communications, Nature, vol. 12(1), pages 1-13, December.
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